I'm Aware That I'm Rare: the phaware® podcast

I'm Aware That I'm Rare: the phaware® podcast is devoted to raising global pulmonary hypertension awareness with dynamic stories from PH patients, caregivers and medical professionals from around the world. Through this series of impactful, insightful and, most importantly, hopeful stories from members of the global pulmonary hypertension community, we hope to further the global #phaware conversation as well as to capture, engage and enable misdiagnosed and undiagnosed PH patients because early diagnosis and treatment can mean the difference between life and death. Topics range from the importance of early diagnosis and global PH awareness, to the impact of clinical trials to how patient support and advocacy are paramount to battling this disease. Learn more about PH at www.phaware.global

Episode 525 - Lisa Mielniczuk, MD 06/17/2025

Jason Weatherald, MD - phaware® interview 524 06/11/2025

Jason Weatherald, MD - phaware® interview 524 Beyond the Clinical Trial: What Real-World Data Means for You. Think only people in clinical trials help advance medicine? Think again. Dr. Jason Weatherald explains how your everyday clinic visits - from walk tests to how you feel - can help shape the future of pulmonary hypertension treatment. Learn what "real-world evidence" is and why your story matters more than you think. I'm Jason Weatherald, I'm from the University of Alberta in Edmonton in Canada. I'm a clinical researcher, I focus on pulmonary hypertension and also on lung transplantation in my practice. I do a lot of research related to patient-oriented ideas and clinical trials. Today I want to talk a little bit about real-world data and real-world evidence and what that means, and why it's relevant in pulmonary hypertension. Real-world data and real-world evidence are related to complementing clinical trials. The way that new treatments get to the marketplace for any disease is through randomized controlled trials. These have to be approved by regulatory bodies, like for instance, the FDA in the United States, or the EMA in Europe. Clinical trials are different than what happens in the real world. When you're running a clinical trial to get a new treatment approved, you want your trial to be successful. The trial has very strict criteria on what type of people can be in it, and they have very strict measurement, and it's very tightly regulated. That's important and that's really good. That's how we know things work under ideal circumstances. However, when a treatment is approved and it starts getting used in the real world, the types of patients that are on a drug or on a device are always different than what's in the clinical trial. We call that real-world populations. So, we also need data and evidence from real-world populations as to how a treatment is actually working in practice. Because the selection criteria for a clinical trial are so narrow and so tight, the results are not always generalizable to the everyday patient in front of you who may be older, who may have other comorbid conditions. They may have a bit of kidney failure, they may be overweight, and sometimes clinical trials don't include those people. So we need other sources of knowing that a drug is safe and also likely to work in the broader population. We call that real-world data. The information that we get from real-world data becomes real-world evidence. That's the type of evidence that we can use to make decisions in clinical practice. Patient reported outcomes can still be used in a clinical trial. Those are any sort of description of how a patient's feeling or their quality of life. It's just what the patients are telling you is going on that can be used in a clinical trial. There are other types of real-world data. I'll give a few examples of what we're talking about with real-world data. After a drug is approved, now most of the regulatory bodies require companies to perform drug registries. These are basically people who are on the drug in the real world and they're collecting information about side effects, because they can look at much larger populations of people who are taking a drug. Maybe rare side effects didn't show up in a clinical trial because there's only 300 or 400 people in the trial. But what happens when 5,000 people are on a drug? So drug registries are one type of real-world data. There's also really powerful other sources that are collected in everyday practice. When you go to the hospital, you might have an electronic medical record, and then physicians and surgeons apply billing codes for all of the services that they provide. That data go to massive databases. So for instance, in the US, the Veterans Affairs has this massive database of people that have sought care, and you can also obtain data from those sources. We call them administrative databases. They may have tens of thousands of patients. You can look at associations and you can try to understand rare side effects or health resource use in the real-world population. So those can be a great source. Patient biometric data is one potential source of real-world data in the future. So you could think about a watch or a Fitbit or an Apple device collecting patients' real-world data on what's happening to them with their step counts, their heart rate in the real world. That is one other potential source that we really haven't capitalized on quite yet in the pulmonary hypertension world. But this has been used in clinical trials already, and it has been used in real-world studies in other diseases in the cardiovascular arena. I think this is one potential area, and you could even consider integrating things like your step count, your heart rate, and your patient-reported outcomes like how do you feel on a given day, multiple days in a month? Using emerging, very sophisticated computer and artificial intelligence approaches, you can get a better picture of how someone's doing day to day from that type of data that might be much more powerful than what we're collecting in clinic visits that are every three or four months apart. Those are just snapshots, and that type of real-world data is more like a videotape. It tells you what's going on all the time. For instance, like administrative databases. I've worked with this in my own healthcare region, and it's all de-identified, it's associated with numbers that could be identified. But when you get the data from, for instance, your health region or your hospital, it has all the identifiers taken out. But it may have things like age, and sex, and sort of how many comorbidities a patient has. The administrative data is de-identified, and it may be under the jurisdiction of a hospital or in Canada where I live in a province, but the drug registries are different. Participants would only get their data into that type of a setting through a normal informed consent process. They would have to sign a consent form to have their data collected. Of course, it is always under the protection of the physicians that are running those studies, and the anonymity and confidentiality of that data is the responsibility of those doctors. None of that identifiable information should be shared with the companies that are asked to run these studies. But it really depends on the setting. One of the things I hear from patients all the time is that they want to participate in research, but maybe they don't want to be in a clinical trial or maybe they don't qualify for clinical trials. This is a way that patients can participate and there's really nothing else that's asked of them. Usually, these drug registries are done as part of routine care. We're only sharing information like, how does a patient feel? What was their walk test? What was their blood test that day? If they have a heart catheterization, what are those numbers? It's a way that patients can feel that they're contributing to knowledge without having to do a bunch of extra things. For many people that's enough. They feel like they're altruistic in some senses, it's people helping the generation of knowledge in a way that they can. The other benefits I think are so that we do learn, how safe are these new treatments? How well do they work in the real world? As researchers, we can ask scientific questions that we can look at the data once it's accumulated over many years to say like, "Well, have you thought about this?" So we can even go to the companies and say, "Well, you should look at this. We're seeing this in the real world. Maybe you should make sure that this isn't the problem." For instance, risks of bleeding or risks of headaches. We might be seeing stuff that wasn't necessarily picked up in a clinical trial, and that can be a red flag that makes us do things differently to protect the safety of patients. There are lots of opportunities to tell patients about different ways they can be involved in research. It really does depend on the individual patient. For instance, at my university, we may be running five or six clinical trials at once, and the criteria for them are different. So we have a cheat sheet and we look at the patient. We always ask them, first of all, "Are you interested in participating?" If not, then the conversation ends there. But if patients are interested and they want to hear more, we do have to spend a bit of time looking at what they would qualify for. For instance, some of these drug registries are only applicable for people who are about to start a new drug. So they're starting drug A, and there's a registry for drug A. It doesn't mean that I would put them in the registry for drug B. So those opportunities only come out often when people are starting new treatments, so they may be few and far between. In terms of clinical trials, it's something I bring up almost every visit and just say like, "Hey, these are the opportunities. Do you want to participate?" If not, we end it there. But people change over time, they might not be eligible for a clinical trial at this visit, but maybe in six months they are because things have changed, or maybe they're not on a certain drug that's excluded, or maybe they're a bit worse and now they qualify for clinical trials. It always adds extra time to the visits, but it's important, I think. One thing I always say is that all of the treatments we have now are only here because of people who participated in trials in the past. Some of those people are no longer with us. That's their legacy, That's the gift of knowledge that they gave to the future generation of people with PH is we learned what works and what doesn't work because of people that participated in trials. I think that's important that people understand that, and acknowledging that it's still not right for everybody to participate in a clinical trial. But what I like about the real world data is that that's just a great example of how people can contribute in other ways. There's other registries. A lot of countries have national registries where patients can sign up. Again, we just collect data from each visit as people come through the clinic, it's just reflecting what happens in the real world, and that's where the name comes from. I'm Dr. Jason Weatherald, and I'm aware that my patients are rare. Learn more about pulmonary hypertension trials at . Follow us on social @phaware Engage for a cure: #phaware Share your story: #phawareMD /episode/index/show/phawarepodcast/id/36413620

Episode 524 - Dr. Jason Weatherald 06/10/2025

Rodolfo Estrada, MD - phaware® interview 523 06/04/2025

Rodolfo Estrada, MD - phaware® interview 523 Dr. Rodolfo Estrada, is a seasoned pulmonologists currently at UT Health San Antonio with significant experience in the management of patients with different forms of pulmonary hypertension. In this episode, Dr. Estrada will discuss the technology and clinical development behind YUTREPIA™ (treprostinil) inhalation powder and why it might be a good option for some PAH and PH-ILD patients. This Special Edition episode is sponsored by . Please see the Important Safety Information following this podcast. The Prescribing Information and Instructions for Use for YUTREPIA (treprostinil) inhalation powder are available at YUTREPIA is approved for the treatment of pulmonary arterial hypertension (PAH; WHO Group 1) and pulmonary hypertension associated with interstitial lung disease (PH-ILD; WHO Group 3) to improve the ability to exercise. Steve Van Wormer: Hi, welcome to I'm Aware That I'm Rare: the phaware podcast. I'm your host, Steve Van Wormer. Today we are here with Dr. Rodolfo Estrada from UT Health in San Antonio, Texas. He’s here to tell us about some exciting happenings that are taking place in the world of pulmonary hypertension and a new therapy that's been brought to market. Dr. Estrada, welcome. Rodolfo Estrada, MD: Thank you so much for having me. It's really great to be here. As you mentioned, I'm Rodolfo Estrada. I'm a pulmonologist at UT Health San Antonio, and there I lead the pulmonary hypertension program. My focus is now on caring for patients with pulmonary arterial hypertension or PAH and advancing research in this area. Steve Van Wormer: Dr. Estrada, it's a very interesting time in the world of pulmonary hypertension. There's a lot of moving parts, a lot of changes taking place in the community. I'd like you to share a little about where we are today. Rodolfo Estrada, MD: You're absolutely right. Even if we’ve made all this progress in PAH, PAH is still serious, and it's a progressive disease that can take a big toll, not only on the patients, but also on caregivers and families. We improved a lot in diagnosing it earlier and managing risk more precisely. We have more treatments available now more than ever, but we still are faced with big challenges. Diagnosis often comes up late, and many patients can’t tolerate the medication or don't have access to advanced therapies when they need them the most. For example, data from the REVEAL registry showed that only about a third of patients had received any form of prostacyclin, and half of those who died had never been started on IV therapy. And we get it. These medications really aren't easy. The side effects can be tough, and depending on how they're given, especially with IV or sub-Q options, there's a real risk of infection or complications from the delivery method. That's why inhaled prostacyclins have become an important alternative. Delivering the medications straight into the lungs helps reduce the side effects that you might get when the drug circulates around the body. But even with inhaled options, there are some hurdles, like needing frequent doses, challenges with breathing patterns, and a ceiling effect, at times, of how much you can increase the dose. Now here's where things get exciting. A new inhaled prostacyclin called YUTREPIA was recently approved. It's a dry powder inhaler that delivers medications using a special technology that’s called PRINT® that makes uniform particles just one micron in size. These particles travel deep into the lungs where they are needed the most. The medication comes in capsules you load into the device, and then you puncture the capsule and take two breaths four times a day. This device requires low effort from the patient, it seems to be well tolerated and easy to use, and hopefully this will help with the adherence of these patients. Steve Van Wormer: It's always an exciting time when there's a new tool in the toolbox for doctors and healthcare providers to offer patients. Can you tell us a little bit about your experience with this clinical trial and the patients that you saw that really led to this approval. Rodolfo Estrada, MD: Sure, happy to. In the Phase 1 study of YUTREPIA, they found that 79.5 micrograms of YUTREPIA gave similar systemic exposure to about nine breaths of nebulized treprostinil. Where in another analysis showed that patients had a greater survival and longer time before starting parenteral therapy when they were on doses greater than this threshold of 79.5 mcg four times a day. Then, there was a study called INSPIRE, which was a Phase 3 open-label, multicenter trial that included about 121 patients. These patients were either started on YUTREPIA or they transitioned from nebulized treprostinil. The main goal was to see if the medication was safe and tolerable, and it was. The study included patients with PAH and WHO Functional Class II and III. Everyone had to be able to walk at least 150 meters and have a good lung function. Most patients were on background therapies, and patients had been diagnosed with PAH for about seven years on average for those who transitioned, and about four years for the ones who were newly started. Those transitioning, had to be on stable nebulized treprostinil for at least three months. They also used an explanatory analysis to look at things like six-minute walk distance and patient-reported outcomes. On average, patients who were new to prostacyclin showed improvement, especially as doses increased, and those who transitioned didn't lose ground in their exercise capacity. Notably, a hundred percent of the patients who transitioned from nebulized treprostinil say they preferred to stay on YUTREPIA after a few months of trying it. Steve Van Wormer: Can you walk us through a little about how the medication was being used in the INSPIRE study or tell us what kind of doses was the study aiming to achieve? Rodolfo Estrada, MD: The way the trial was done is patients were started on 26.5 micrograms four times a day, with weekly increases up to a maximum of 212 micrograms four times a day. By two months, 78% of the transition group and 71% of the treatment-naive group were above the key threshold of 79.5 micrograms four times a day. At one year, 40% reached a dose equal or greater to 132.5 micrograms or what's the equivalent of about 15 breaths of nebulized treprostinil. In the open label extension, about one third of patients reached a dose equal or greater than 159 micrograms, which is about 18 breaths of nebulized treprostinil. Even at two years, there were patients who reached up to 238 micrograms, which is around 27 breaths of nebulized treprostinil. In our center, we had a couple of patients in the trial who reached some of the highest doses, even in a woman in her 70s, which again, trial patients may not necessarily represent the real world. They tolerated it well and stayed at low risk longer really without needing additional therapy. Steve Van Wormer: So in these trials, what were some of the potential side-effects that patients experienced as they went through the trial? Rodolfo Estrada, MD: That’s a good question. The side effects were similar to other inhaled prostacyclins like cough, throat irritation, headaches and dizziness. These were mostly mild to moderate and tended to show up more in the treatment-naive group, as you would expect. Importantly though, at one year, no patient in the transition group had to stop the medication because of cough, and only 2 patients in the treatment-naive group had to. About 10% left the study due to side effects, but none of them were serious adverse events that were linked to the medication. Steve Van Wormer: So you had this successful trial and that’s led to a successful approval. Now that there's a new drug in your toolbox, what happens when a new therapy comes into play into the real world versus when it’s used in a trial group of patients? Rodolfo Estrada, MD: When we're doing trials, we're trying to answer a very specific question. And to be able to answer that specific question, we try to narrow the population that we can get the answer for. What happens in real world is that then the exposure of whatever intervention we did, medication or whatever else we would do, would be then offered to a broader range of profiles. And then things that we may have not picked up on trials, then we pick it up in the real world, sometimes great, sometimes not as great. When you do a trial and it's a specific number of patients, usually a smaller number of patients than real world, as you get more exposure in the real world, we learn new things that we didn't know about it before. Steve Van Wormer: So what's great here in summary, doctor, is that we have a whole host of options for PAH patients, including YUTREPIA here, but also exciting for the PH-ILD population that has formerly only had one option. So, when a second option, a new option is laid out, how does that change the playing field for both of those subsets of patients? Rodolfo Estrada, MD: Yeah, it's a great point. We now have another inhaled option with YUTREPIA, which is now approved for not only PAH but also PH-ILD. It's a convenient dry powder inhaler that requires low effort from the patient, and it seems to be well tolerated and easy to use, which may help with the patient's adherence. From what we've seen, it offers a chance to deliver medication deep into the lungs, hit high doses, and potentially improve outcomes for patients who may not tolerate or access other forms of the medication. Steve Van Wormer: I really appreciate you being here today, doctor, to tell us about this exciting news for patients, exciting news for the healthcare providers like yourself, and thankfully to sponsors like Liquidia for the work they're doing to better the lives of patients with PAH and PH-ILD. Rodolfo Estrada, MD: Thank you for having me, Steve. It’s really been a pleasure talking to you about this today. And for the patients listening, while it is always exciting to have a new medication available in this space, it is very important that you discuss to the specific therapy plan that is best for your with your specific therapy providers and teams. My name is Rodolfo Estrada, and I'm aware that my patients are rare. Please see the Important Safety Information in the show notes. The Prescribing Information and Instructions for Use for YUTREPIA are available at Learn more about the Learn more about pulmonary hypertension trials at . Engage for a cure: #phaware Share your story: Like, Subscribe and Follow us: . #phawareMD #PHILD IMPORTANT SAFETY INFORMATION Before you take YUTREPIA, tell your healthcare provider about all of your medical conditions, including if you: Have low blood pressure Have or have had bleeding problems Have asthma or chronic obstructive pulmonary disease (COPD) Are pregnant or plan to become pregnant. It is not known if this product will harm your unborn baby Are breastfeeding or plan to breastfeed. It is not known if this product passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby during treatment Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. YUTREPIA and other medicines may affect each other. Especially tell your healthcare provider if you take: Medicines used to treat high blood pressure or heart disease Medicines that decrease blood clotting (anticoagulants) Water pills (diuretics) Gemfibrozil (Lopid®) or rifampin (Rimactane®, Rifadin®, Rifamate®, Rifater®) What are the possible side effects of YUTREPIA? This product can cause serious side effects, including: Low blood pressure (symptomatic hypotension). If you have low blood pressure, this product may lower your blood pressure more. Bleeding problems. This product may increase the risk of bleeding, especially in people who take blood thinners (anticoagulants). The most common side effects of YUTREPIA are cough, headache, throat irritation and pain, nausea, reddening of the face and neck (flushing), fainting or loss of consciousness, dizziness, diarrhea, and shortness of breath. Like other inhaled prostaglandins, you may have trouble breathing after taking YUTREPIA because it may cause the muscles around your airway to tighten (bronchospasm). These are not all the possible side effects. Call your doctor for medical advice about side effects or if you have trouble breathing. You may report side effects to the FDA at or call 1–800-FDA-1088. The risk information provided here is not comprehensive. To learn more about YUTREPIA, talk with your healthcare provider. Please see and . For additional information, call 1–888–393–5732. /episode/index/show/phawarepodcast/id/36789935

Episode 523 - Rodolfo Estrada, MD 06/03/2025

Episode 523 - Rodolfo Estrada, MD Dr. Rodolfo Estrada, is a seasoned pulmonologists currently at UT Health San Antonio with significant experience in the management of patients with different forms of pulmonary hypertension. In this episode, Dr. Estrada will discuss the technology and clinical development behind YUTREPIA™ (treprostinil) inhalation powder and why it might be a good option for some PAH and PH-ILD patients. This Special Edition episode is sponsored by . Please see the Important Safety Information following this podcast. The Prescribing Information and Instructions for Use for YUTREPIA (treprostinil) inhalation powder are available at YUTREPIA is approved for the treatment of pulmonary arterial hypertension (PAH; WHO Group 1) and pulmonary hypertension associated with interstitial lung disease (PH-ILD; WHO Group 3) to improve the ability to exercise. Please see the Important Safety Information in the show notes. The Prescribing Information and Instructions for Use for YUTREPIA are available at Learn more about the Learn more about pulmonary hypertension trials at . Engage for a cure: #phaware Share your story: Like, Subscribe and Follow us: . #phawareMD #PHILD IMPORTANT SAFETY INFORMATION Before you take YUTREPIA, tell your healthcare provider about all of your medical conditions, including if you: Have low blood pressure Have or have had bleeding problems Have asthma or chronic obstructive pulmonary disease (COPD) Are pregnant or plan to become pregnant. It is not known if this product will harm your unborn baby Are breastfeeding or plan to breastfeed. It is not known if this product passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby during treatment Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. YUTREPIA and other medicines may affect each other. Especially tell your healthcare provider if you take: Medicines used to treat high blood pressure or heart disease Medicines that decrease blood clotting (anticoagulants) Water pills (diuretics) Gemfibrozil (Lopid®) or rifampin (Rimactane®, Rifadin®, Rifamate®, Rifater®) What are the possible side effects of YUTREPIA? This product can cause serious side effects, including: Low blood pressure (symptomatic hypotension). If you have low blood pressure, this product may lower your blood pressure more. Bleeding problems. This product may increase the risk of bleeding, especially in people who take blood thinners (anticoagulants). The most common side effects of YUTREPIA are cough, headache, throat irritation and pain, nausea, reddening of the face and neck (flushing), fainting or loss of consciousness, dizziness, diarrhea, and shortness of breath. Like other inhaled prostaglandins, you may have trouble breathing after taking YUTREPIA because it may cause the muscles around your airway to tighten (bronchospasm). These are not all the possible side effects. Call your doctor for medical advice about side effects or if you have trouble breathing. You may report side effects to the FDA at or call 1–800-FDA-1088. The risk information provided here is not comprehensive. To learn more about YUTREPIA, talk with your healthcare provider. Please see and . For additional information, call 1–888–393–5732. /episode/index/show/phawarepodcast/id/36648690

Marc Simon, MD - phaware® interview 522 05/27/2025

Marc Simon, MD - phaware® interview 522 Decoding Pulmonary Hypertension: Echo and Cath Insights for Pulmonologists. Dr. Marc Simon shares his expertise on diagnosing pulmonary hypertension, emphasizing echocardiographic markers, right heart catheterization pitfalls, and risk stratification with the H2FPEF score. His insights help clinicians refine their diagnostic approach for better patient outcomes. This Special Edition episode is sponsored by . View PDF Slides . I’m Marc Simon. I am a cardiologist and a heart failure specialist at UCSF in San Francisco. I direct the pulmonary hypertension program there. I moved there about four years ago now in 2021 for this opportunity from the University of Pittsburgh where I was on faculty for about 15 years, taking care of pulmonary hypertension patients within our cardiology section. Very similar to UCSF, in a very multidisciplinary group with both pulmonologists and cardiologists. It’s really been a really wonderful experience over these past 20 or so years. I’ve done a lot of research over the years in terms of right ventricular function and adaptation in pulmonary hypertension, really ranging from imaging with echocardiography as we’ll talk about today, MRI, some CT scans, as well as integrating with hemodynamics to understand cardiovascular physiology of the right ventricle, as well as some translational work to try to understand the biomechanics of the right ventricle, which is an exciting and ongoing story. Finally, some drug development over the last eight years or so. That’s been really exciting. As you know, with pulmonary hypertension, we’re very fortunate to have so many drugs available to treat patients, so it’s been a wonderful career so far. It’s a really interesting time in pulmonary hypertension. There’s continued to be a lot of drug development going on with a lot of focus now on ILD and related pulmonary hypertension, which is particularly exciting in a space where prior we had no therapies. In that context, I’ll be talking to you today about echocardiography and right heart catheterization for the pulmonologist. This is a talk I gave recently at the California Thoracic Society. I really look forward to presenting some of this information today as a cardiologist, I guess, talking to the pulmonary community and folks involved with pulmonary hypertension, and potentially patients as well, getting some insights as to some of the testing that we do to better understand disease and diagnose. When I think about echocardiography for pulmonary hypertension, I’m kind of thinking really sort of broadly speaking in three different contexts that I find to be really helpful to think about this. One is echocardiography, of course, useful for estimating a pulmonary artery pressure. That’s what most people will think about and that can be really useful. That echocardiography is a great screening test for the pulmonary pressure. Then, I really like to come at the echocardiogram in terms of trying to end up at a phenotype that is either right-sided or left-sided. I think that’s a really nice context to think about the echocardiogram as you’re going into this. By right-sided, I mean, sort of a RV failure type phenotype, and that’s really what we’re talking about for pulmonary hypertension, pre-capillary pulmonary hypertension. As opposed to left-sided, thinking about really ending up being group two pulmonary hypertension and all the causes thereof related to heart disease and things like this. You’ll hear me talking about right versus left-sided phenotyping as we go through some of these highlights of echocardiography for pulmonary hypertension. Finally, it’s always important not to forget with the echocardiogram, that’s a great screening test for congenital heart disease. So, of course, that’s a very unique and specific cause of pulmonary hypertension. A lot of times, you’ll get your first inkling that this may be what’s going on, on the echocardiogram. I like to start off by highlighting a study that came out a few years ago, actually, out of Australia by a Strange and colleagues, it was published in Heart in 2012. They just looked at all echocardiograms that had an estimated pulmonary artery pressure greater than 40 millimeters of mercury. All of those coming through the echo lab, out of those, two-thirds ended up being left heart disease. So, the vast majority of what we’re seeing that’s referred to us as an echocardiogram that might be suggestive of pulmonary hypertension with an elevated estimated pulmonary artery systolic pressure really ends up being left heart disease. So, again, really important to keep this in mind. Only 2.7% of those patients ended up having pulmonary arterial hypertension. About 2% had chronic thromboembolic pulmonary hypertension and 9% had lung related disease. The 2022 European Guidelines think about echo screening in terms of three contexts. This is the ventricles, the pulmonary artery and the inferior vena cava and the right atrium. Showing signs from at least two of these can alter your level of a probability of pulmonary hypertension. When we’re talking about the ventricles, there’s a couple of points. One is the right ventricle larger than the left ventricle. This can be as simple as a basal diameter in which the right side is greater than the left side. Along with this can be flattening of the interventricular septum. Those two points right ventricular enlargement, RV size greater than LV size and flattening of the septum falls into that context of a right-sided phenotype that I was mentioning. Then, talking about the TAPSE, tricuspid annular plane systolic excursion or that ratio of that to the systolic pulmonary artery pressure being less than 0.55 millimeters per of millimeter of mercury. I’ll talk a little bit about what that particular measure is. Some people like it, some people don’t. TAPSE is a measure of right ventricular function. Of course, systolic pulmonary artery pressure is the pressure afterload that the right ventricle sees. In terms of the pulmonary artery, a dilated pulmonary artery, like I’m sure a lot of pulmonologists are really keyed into. In fact, I usually receive several referrals a month based on an enlarged pulmonary artery seen on a CT scan and is this related to pulmonary hypertension. So, the enlarged pulmonary artery can also be seen on echocardiography. That can be a real phenotype that keys you into precapillary pulmonary hypertension. Also, is the pulmonary artery acceleration time. This turns out to be my favorite, favorite term in echocardiography. I’m going to talk to you a bit more about that. But essentially, it’s the flow out of the right ventricle through the pulmonary valve into the pulmonary artery, and you can measure the onset of that flow to the peak velocity. If it’s less than 105 milliseconds, call it 100 milliseconds, it’s easier to remember. That is a good cut point to say, “Hey, maybe this patient has significant pulmonary hypertension.” Again, I’m going to talk about that a lot more. Then, a dilated inferior vena cava, significant for right ventricular volume overload. Right ventricular failure, a high central venous pressure, for example, and a dilated right atrium. These are other signs of that right-sided phenotype again. Again, if you have signs from at least two out of these three categories, it can be highly significant for pulmonary hypertension. A lot of people are familiar with this concept of the large dilated right ventricle and right atrium, the flat septum, the pericardial effusion that a lot of times can be seen in pulmonary arterial hypertension. We’ll have a to some images of this if that’s helpful for folks. Let’s talk a little bit more about my favorite echocardiographic measure, the pulmonary artery acceleration time or PA acceleration time or sometimes called PAAT. If you’re in the parasternal short axis view at the valvular plane, for those of you who have looked at echocardiograms, that view where you see the cross section of the aortic valve right in the center there and on the left side of the screen is the tricuspid valve with the right ventricular outflow tract arching over the center of the top of the screen over the aortic valve, and then the pulmonic valve on the right side of the screen leading out to the main pulmonary artery. That classic view of the basal cut of the parasternal short axis view. When we look at the flow on that right side of the screen, so from the right ventricular outflow tract out through the pulmonary valve into the main pulmonary artery, we can look at the Doppler velocity of the blood flow in that direction. That flow is what we call the ejection from the right ventricle out into the pulmonary artery. Again, we can measure the onset of that flow to peak velocity because in Doppler terms, we’re measuring velocity. So, the peak of that velocity, and this is really sort of a U-shaped type of velocity envelope as we call it. So, the onset to the peak of that U, the X axis is time, and we’re measuring that in milliseconds, so, that time is the pulmonary artery acceleration time. As it turns out, it’s highly sensitive to pulmonary pressure and it gets shorter as pulmonary pressure is elevated. So, it’s inversely related to pulmonary pressure. I quiz the trainees a lot on this. Why would that be? Why is it an inverse relationship? Well, it turns out this is related to cardiovascular physiology. So, if you think back to medical school, the idea of isovolumic contraction of the ventricle, because after the ventricles filled, you have to develop enough pressure to overcome the pressure that you’re pumping out against, in this case the pulmonary valve and the pulmonary artery. So, ventricular pressure during the initial contraction phase has to develop enough pressure to exceed pulmonary artery pressure. Then, the pulmonic valve opens once that pressure gradient is exceeded. Once the valve opens, then we have ejection. After ejection is complete in terms of enough volume has been ejected from the ventricle, that ventricular pressure then starts to fall. When right ventricular pressure falls below pulmonary artery pressure, the pulmonary valve will close again, and then you’ll have isovolumic relaxation until pressure falls below the right atrial pressure. Then, filling begins through the tricuspid valve. If you think about pulmonary hypertension, the higher the pressure in the pulmonary artery, the more that the right ventricle has to overcome in order to eject its volume. If you think about the cardiac cycle being fixed, then the higher the pressure, the more time the ventricle is going to spend in isovolumic contraction to develop more pressure to then overcome the pressure in the pulmonary artery and eject. So, there’s less relative time in ejection. So, that means, all of the blood flow out of the right ventricle into the pulmonary artery has to occur in less time. You can imagine that really squashes down, that narrows down the velocity window of ejection. So, the time from onset of flow to peak flow becomes much narrower. That’s that inverse relationship between pulmonary artery acceleration time and pulmonary pressure. There was a nice paper published in 2011 actually, in the Journal of the American Society of Echocardiography, first author Yared that looked at the PA acceleration time and created an equation linking it to estimated pulmonary artery systolic pressure. I really like this because you can then relate directly the PA acceleration time in milliseconds to pressure. When I calculate that out, if you have a PA acceleration time of 150 milliseconds, that calculates out by this paper to 32 millimeters of mercury. So, not too bad. If the PA acceleration time, then decreases to 100 milliseconds, remember this is the cut point for the European Guidelines, 105 milliseconds, very close. That calculates out to an estimated PA systolic pressure of 50 millimeters of mercury. You can see right there, you’re at a significant pressure in a range where we would really say, “Oh, that might be someone we should look at further.” PA acceleration time of 50 milliseconds, which I’ve seen quite frequently, calculates out to an estimated PA systolic pressure of 79 millimeters of mercury (mm Hg). Really, that’s significant pulmonary artery pressure. Beyond just being able to have another measure of estimated pulmonary artery systolic pressure, there’s a couple other things I like about this. One is that it’s not just numeric. I’ve told you a lot about the numerics and the physiology, but that can be a little bit complicated. There’s also a qualitative version of this, which is, if you’re looking at that velocity envelope, it’s is called mid-systolic notching. Along with a steepening of the velocity going out into the pulmonary artery, on the backside of that nice U-shaped (usually) curve, you’ll get a notching. This can look like a W or an M. It’s very obvious in many cases. That qualitative sign also goes along with significantly elevated pulmonary pressure. A lot of times actually, when I’m reviewing an echo where I don’t have that measure already made for me and I don’t have the ability to measure it on my own on the fly, I’ll make a note to myself, “Oh, this patient has mid-systolic notching or this patient does not have mid-systolic notching.” Again, that to me is another one of those check boxes for is this a right-sided phenotype? There is mid-systolic notching. Or a left-sided phenotype, there is no mid-systolic notching? There’s a few examples of this that will be in the . The final reason, I really like pulmonary artery acceleration time is the incomplete tricuspid regurgitation jet. I’m sure everyone’s seen an echocardiogram report where it says there’s an incomplete TR jet. Therefore, we cannot estimate pulmonary artery systolic pressure. That occurs in really upwards of 25% of echoes. So, it’s quite substantial. When this occurs, almost always there’s a viable PA acceleration time or RV outflow Doppler signal that’s in the echocardiogram and can be pulled out. Even in these indeterminate echocardiogram reports, many times, we can actually come up with an actual estimated PA pressure using the PA acceleration time. The other things I like to look at with the echocardiogram are right ventricular function. We’ve talked a little bit about right ventricular structure, is it dilated or not? We’ve talked about the estimated PA pressure and the related lauded pulmonary artery acceleration time. Finally, let’s talk about right ventricular function. So, again, diminished right ventricular function will fall into that right ventricular phenotype echocardiogram versus normal right ventricular function, maybe, there’s not a significant pulmonary hypertension. There’s a number of metrics now out there to help evaluate right ventricular function. One of the main ones that I would say is reported on more echoes than not these days is TAPSE. Probably, a lot of people are aware of this. It’s not a new measure at this point, but it is related to the longitudinal motion of the right ventricle. This is a large portion of right ventricular function, not all of it, but a large portion of right ventricular function. We can measure how much motion is in that tricuspid plane just by measuring in one dimension the motion in centimeters or millimeters. The more motion there is, the more normal the right ventricular function is. There are various cut points between 2.0 centimeters, that’s 20 millimeters and 1.6 centimeters or 16 millimeters. Why is this so important? There are a number of papers that had come out thathave associated related diminished TAPSE with poor outcomes. There’s a lot of prognostic value with this. It’s highly correlated with pulmonary pressure. So, it’s a really useful term to have in our reports. This is one of several that I’ll try to write down. The next one is what’s called right ventricular S’. S stands for systole or systolic contraction. The prime (‘) indicates that this is a tissue Doppler measure. So, we’re actually measuring the velocity of the tissue in this case and not the blood. If we look kind of in a similar spot to where we look for TAPSE on the lateral wall at the basal level near the tricuspid valve, we can put a cursor there and we can measure the velocity of the myocardium of (the muscle of the right ventricle). A cut point for this is about 10 centimeters per second. If you’re above this, you have vigorous contraction. If you’re below this, you have right ventricular dysfunction. Again, another measure that is in many reports these days, I’ll add it along with TAPSE to sort of complete this story. I kind of find that with right ventricular function. Remember, the right ventricle is a very complex three-dimensional structure. Echocardiography is a one-dimensional or two-dimensional modality of imaging. You need to provide multiple views, multiple metrics to really best describe right ventricular function I find. I don’t think any one measure is good enough to really give you a full picture of right ventricular function. The more of these you can sort of put together the better. So, we have TAPSE. We have right ventricular S’. Then, there’s also strain, right ventricular strain. In this case, we’re looking at image processing of the right ventricle, and we can look at the shortening of the myocardium from one frame to the next as the right ventricle contracts, and the percentage shortening is correlated with right ventricular function. We can look at the free wall of the right ventricle. We can look at the septum. We can average all of those together. More often than not, I like to look at the mid right ventricular free wall, a shortening of minus 20% or so in absolute terms, more, if you want to talk about it or in negative terms, more negative is normal function, and less than that is abnormal. Then, more recently, there have been some attempts to put together several of these metrics to help us guide is this more of a right ventricular phenotype, pulmonary hypertension, precapillary pulmonary hypertension phenotype, or more of a left-sided phenotype or Group 2 pulmonary hypertension phenotype. One of these is the VEST echo screening tool, which looks at three parameters. One is the mitral E/e’. I haven’t talked about this too much, but this is a measure of left ventricular diastolic function. I put left ventricular diastolic dysfunction into that left ventricular phenotype category. You can have a whole lecture just on that alone. But another probably good point to look at and keep an eye open for on our echocardiograms is simply has there been a mention of a grading of left ventricular diastolic function? If there is, that might be one of these warning signs that maybe we’re dealing with a left-sided phenotype. But in any event, the VEST echo screening tool looks at the mitral E/e’, looks at left atrial size. We talked a little bit about right atrial size. A dilated right atrium, a significant for precapillary pulmonary hypertension, that right-sided phenotype. A dilated left atrium is a big marker of left-sided disease. Then, finally, septal flattening. We’ve talked about that before too. So, more to come on that measure (VEST echo screening tool), I think, as there’s more work being done on it. With that, I’ll switch gears to right heart catheterization and talk a little bit about that. So, with right heart catheterization, as... /episode/index/show/phawarepodcast/id/36058850

Episode 522 - Marc Simon, MD 05/26/2025

Jean M. Elwing, MD - phaware® interview 521 05/20/2025

Jean M. Elwing, MD - phaware® interview 521 Dr. Jean Elwing, a leading expert in pulmonary hypertension, discusses groundbreaking advancements in treating pulmonary hypertension associated with interstitial lung disease (PH-ILD). For years, patients with this condition had limited treatment options beyond oxygen therapy and symptom management. However, recent studies have introduced new hope, showing improved patient outcomes and quality of life. Dr. Elwing emphasizes the importance of early diagnosis, ongoing research, and clinical trial participation in pushing the field forward. This Special Edition episode is sponsored by and . My name is Dr. Jean Elwing. I'm a Professor of Medicine and I'm the Director of the Pulmonary Hypertension Program at the University of Cincinnati. I've been there about two decades now, really focused the entire time on the care of patients with pulmonary hypertension and related illnesses. Over that time, we've built a large practice of pulmonary vascular disease related conditions and are trying to do our best to improve the lives of people we take care of. Today, I'd like to talk to you about interstitial lung disease and pulmonary hypertension. The type of pulmonary hypertension that develops in the setting of lung disease is called hypoxic pulmonary hypertension or Group 3 disease. This can happen in different types of lung diseases, including interstitial lung disease and COPD to name a few. But we're going to concentrate today on interstitial lung disease and its associated pulmonary hypertension. It's a very exciting time for patients with pulmonary hypertension associated with interstitial lung disease, because we now are making progress in this area. Five years ago, we knew that pulmonary hypertension was associated with this condition, but we didn't have a lot we could do to focus on the pulmonary hypertension except watching, giving oxygen, monitoring right heart failure symptoms and treating them and referral to transplant in appropriate patients. But recently, there's been more of an interest in assessing medications for the treatment of the pulmonary hypertension that occurs in the setting of interstitial lung disease. We've learned over time that this is not easy to treat. We've tried multiple medications without successful outcomes. In small studies, case series, but more recently we studied inhaled treprostinil for the treatment of interstitial lung disease and pulmonary hypertension and found that this was an intervention, a therapy that could improve outcomes. So for the first time, we have hope that we can change the outcome in this disease, slow it down, pump the brakes, give patients a little more time, and hopefully feel better, do more during that time. We would love to have a cure for it. We would love to be able to say we are turning the dial back to zero. We're not there yet, but there's a lot of interest now. There's new studies coming, new therapies being looked at. I have a lot of hope that we're going to be able to make progress here. Interstitial lung disease is a condition where you have inflammation and scarring in the lungs. It can happen from multiple different reasons. It could be related to exposure, it could be related to underlying autoimmune disease or multiple other factors can contribute. But at some point in patients' lives, they may develop a new problem associated with that, and that's pulmonary vascular disease and pulmonary hypertension. At one point, we really thought that that only happened when the disease was quite advanced, but we've learned over time, it can happen at any time in the course of a patient with interstitial lung disease. Once it develops, it's associated with a lot of different things, including more oxygen requirement, less exercise tolerance, more exacerbations, and a shorter life expectancy. It is a trigger for us to think about, “Hey, what can we do to try to slow this down?” We try to optimize the interstitial lung disease, manage everything that's contributing, take away any potential triggers for worsening, manage infections quickly, so that doesn't cause a worsening event requiring hospitalization and additional intervention. But the pulmonary hypertension piece is the part that, as I mentioned, did not have an intervention previously. Now, when we see patients that have signs that their pulmonary vascular disease may be progressing, like more oxygen, lower exercise tolerance, more fatigue, right heart failure symptoms, we look, we obsess with an echocardiogram. We proceed to right heart catheterization to evaluate for the possibility of true elevated pulmonary pressures or pulmonary hypertension. Then, we offer treatment to the appropriate patients. That treatment's not easy, it's an inhaler, four times a day, but as I mentioned, we've learned that it can be very helpful to patients based on the INCREASE trial, where we saw that patients were able to ambulate further. It reduced stress on the heart based on a lab marker called BNP, and it reduced worsening. So this is just the tip of the iceberg. We're just starting this journey with pulmonary hypertension in the setting of interstitial lung disease, but at least now we have a starting point. As I mentioned, there is a therapy available and approved for patients with interstitial lung disease and pulmonary hypertension, and we're working on others through research. What I have to remind everyone is to say thank you to all those individuals who participate in . They give us options. Without their efforts and all the work they do to participate in trials, we wouldn't have new medications. So I just encourage you, if you are the right person that meets criteria for a to consider it. Look at the options. See if that might be something you would like to explore. Not only could it be something that would benefit you, potentially, it also has the opportunity to impact many other individuals affected by this condition. Pulmonary hypertension has so many faces, and for so long we've really concentrated on pulmonary arterial hypertension Group 1 disease. But now, I'm so happy to say that we are getting options for therapies that are impacting outcomes in many groups of pulmonary hypertension, including patients with lung disease. We have a long way to go. We've only touched the tip of the iceberg on this. ILD individuals have an option for treatment. We have many other conditions that of course, over time we hope to better understand and hopefully with that understanding be able to impact the development of pulmonary hypertension and control of the condition to allow patients to have longer, fuller, and more active lives. Thank you so much for listening. My name is Dr. Jean Elwing, and I'm aware that my patients are rare. Learn more about pulmonary hypertension trials at . Engage for a cure: #phaware Share your story: Like, Subscribe and Follow us: . #phawareMD #PHILD #PHOCUSstudy #PROSERAstudy /episode/index/show/phawarepodcast/id/35841670

Episode 521 - Jean M. Elwing, MD 05/19/2025

Seth Hall, MBA, RRT - phaware® interview 520 05/13/2025

Seth Hall, MBA, RRT - phaware® interview 520 Seth Hall, MBA, RRT, takes listeners on a journey through the past, present, and future of inhaled therapies. Discover how these treatments have evolved, the life-changing benefits they offer, and the revolutionary technologies that could redefine outcomes for PAH and PH-ILD patients. This Special Edition episode is sponsored by . My name is Seth Hall. I started my career as a critical care respiratory therapist in Washington D.C. Over the years, I traveled around the country working at various academic centers; Duke and Stanford and Columbia, and several others. I was just like a sponge. That experience just allowed me to keep learning, growing, and luckily, I was able to surround myself with some really great mentors. I landed in respiratory and took care of a lot of patients with pulmonary hypertension in my past, throughout my years working clinically. I'm actually from a very small coal mining community in West Virginia. Some of my current research I do independently from work also revolves around addressing barriers and access to care with patients with cardiopulmonary illnesses, specifically in rural communities. I'm very passionate about advocating for patients with cardiopulmonary diseases and illnesses, and very lucky and fortunate to have been involved in the care of PH patients in my past. Currently I work as a medical science liaison or MSL at , and we strive to bridge the gap between that clinical research and finding solutions to unmet needs for patients. We really help bridge that gap of clinical research and the scientific discussion and exchange. With my respiratory background, something I've always been interested in is just the evolution of inhaled therapies and how we've got to where we are now with managing some of these complex respiratory diseases. Last year, we published a piece in Respiratory Therapy: A Journal of Pulmonary Technique where we talked about just that. We looked at the historicity benefits, future potential options for inhaled therapies, and we really focused on emerging technologies. We wanted to really focus on how targeted drug delivery could potentially transform care for millions of patients. Looking back, it actually goes back pretty far. Almost 4,000 years, actually. Some early medical practices that emerged in India over 2000 BC, where they looked at inhaled therapies for opening up the lungs and what we refer to as bronchodilatory properties. Then, around 1500 BCE, Egyptians were looking at inhaling vapors and other plant medicinal purposes for other respiratory relief. Even when you look back at ancient Greeks under Hippocrates, they developed one of the earliest actually medical devices, and that was simply just a pot with a reed and you inhaled medicines. These vapors were therapies for patients. Then, these practices, which we see all over the world, were found in Central and South America and Africa. They were the early foundation for what we're doing today in the development of modern inhalation therapy. This evolution of inhaled therapies, it really just reflects the ongoing innovation to provide more solutions for people around us. To fast-forward, we now have nebulizers. These emerged around the 19th century. These delivered medicine via an aerosol. Think of a liquid droplet. It can be very effective, but sometimes can be quite cumbersome, with parts and pieces due to the size and assembly. You can imagine being a patient and trying to travel with this. If you have an active lifestyle, it might not be practical for daily use. Then, fast-forward a little bit more looking into around the 1950s, metered-dose inhalers came around or MDIs. These were a good alternative to nebulizers, however, they really require precise coordination. What I mean by that is essentially the patients have to actuate the drug and then time it with their inhalation or with breathing in. You can imagine how for some patients this could be a challenge. Now we have dry powder inhalers or DPIs, which emerged around the 1990s, that really addressed some of those limitations that we spoke to with earlier devices. They're simple to use, they're portable, but they're actually breath-actuated. In order to disperse the medication and get them where they needed to go, the patient just takes in a deep breath. This really helped out with usability and patients adhering to medicine, because we know if we make it easier for patients to take their medicine, they're more likely to be compliant with their treatments. Direct lung delivery has proven itself to be the cornerstone for effective treatment, especially for respiratory diseases. By giving the medication directly to the lungs, we are bypassing potential systemic side effects. The intended site of action is in the lungs, that's where we're delivering the medicine. Whereas, if you're getting medicine via orally or IV administration, you're now exposing other parts of the body to that medication, as well. Essentially, the localized drug delivery can help enable lower therapeutic dosing. You can use less drug. It provides a very rapid onset, you're going to where the drug needs to be. This targeted delivery for symptoms and all different types of diseases, you might have bronchoconstriction or vasoconstriction, inflammation. Essentially, you're just targeting exactly where the drug needs to go. DPIs have become a preferred choice for many respiratory diseases due to their portability, their ease of use, patient adherence, making it easier for patients to take their medicine. Unlike the metered-dose inhalers or the MDIs we just talked about, DPIs don't require that precise timing. They don't have a propellant. They don't require a spacer to take the medication. Then, their compact design makes it ideal for active patients on the go that are out and about with their families. From a functional perspective, DPIs are breath-actuated. What that means is essentially it utilizes the energy of their own breath or their inhalation to overcome these cohesive forces that are holding the powder together. That helps to break up the particles. That process of breaking up the particles is referred to as de-agglomeration. So, the way that devices are engineered with DPIs, there's an internal resistance, and this resistance within the device provides a mechanical assistance with that de-agglomeration, and that helps with the distribution of the drug particles to the lungs. Despite these advancements that we've had in respiratory treatments, inhaled drug deliveries still remains pretty poor. For instance, in the case of DPIs, what's currently available, patients with restrictive lung diseases might have a limited ability to use some of these higher resistance inhalers that the inhaler needs to break apart the medication. This essentially inhibits the complete particle de-agglomeration for the patients. Then, we know that when there's inadequate de-agglomeration that the dry powders are going to have more variability in their particle sizes. Some of these larger particle sizes tend to deposit more in the upper airway, which we refer to as the oropharynx, which can lead to patients having to cough. Apart from increasing the potential upper airway irritation and the cough, you also have insufficient delivery of medication now to the lower airway where you want the drug to be. This could potentially result in increased systemic absorption and creating additional tolerability issues and challenges for patients trying to take their medicine. So, a critical advancement would be if in the inhalation delivery space, we could make these particles uniform in their physical and chemical flow properties that might help resist this clumping together. Some of these advancements would really enable the use of lower resistance inhalers, because it wouldn't necessitate that you need to de-agglomerate with a high resistance. That means less effort that the patient requires for them to take their medication. One novel formulation approach that has the potential to significantly advance the manufacturing of DPIs is something referred to as by Liquidia. PRINT®, which stands for particle replication and non-wetting templates, it actually originated in the electronics industry with microprocessors. Clinically, it's been studied though in vaccines and antivirals, ophthalmology, and even in topicals. Currently, it's of high interest in the inhaled therapy space. PRINT® Technology offers the unique ability to control for inhaled particle size and shape, giving a higher degree of confidence that the drug is reaching the targeted lung region where it needs to go. It's essentially a mold technology. Think of ice cubes in an ice tray. They all look the same shape and size. That's what PRINT® does but on a micro level, with the actual formulation of these DPIs. The geometric precision and shape of these particles can potentially significantly enhance the aerosolization and potentially allowing these PRINT® formulations to be compatible with a diverse array of DPI devices, whether you have low or high resistance. Now, you can tailor the medicine specifically to the pathophysiology of whatever respiratory condition you're trying to treat. PRINT® Technology is designed to ensure that you have the homogeneity of the particle, concentration, and it's a critical factor in maintaining consistent dosing. We know that consistent dosing leads to therapeutic efficacy. This uniformity, it potentially mitigates the variability in drug delivery for patients. As a respiratory therapist, I'm actually really excited. I think the evolution of device technology, along with advancements in drug formulations, holds great promise in improving pulmonary drug delivery. I mentioned formulation, so I'll just grow on that a little bit too. We have emerging liposomal formulations that are being studied right now in clinical trials that might also mark a significant milestone in this ongoing effort. Liposomes are essentially nanoscale lipid vesicles that surround the medication or what we refer to as encapsulate. That's to help extend the release profile of the drug. It also can help enhance the stability, and now, you have a reduction in the dosing frequency required by the patient. These innovations could potentially provide sustained therapeutic benefits for a wide variety of respiratory illnesses, reducing the burden of the frequent dosing for the patients, and potentially improving patient adherence by making it easier on them. All in all, the development of inhaled therapies really demonstrates our relentless pursuit, striving for more tolerable, patient-friendly just more options in the treatment of many conditions in the respiratory space. Innovations such as PRINT® Technology and liposomal formulation really underscores the commitment to optimizing drug delivery for some of these really challenging conditions. As research continues and we have more data, the future of inhaled therapies holds immense potential to really address some of these unmet needs, and most importantly, improve the quality of life for patients all over the world. My name is Seth Hall, and I'm aware that the patients we serve are rare. Learn more about pulmonary hypertension trials at . Engage for a cure: #phaware Share your story: Like, Subscribe and Follow us: . #phawareMD #PHILD #PRINTTechnology /episode/index/show/phawarepodcast/id/36058550

Episode 520 - Seth Hall, MBA, RRT 05/12/2025

Richard Channick - phaware® interview 519 05/06/2025

Richard Channick - phaware® interview 519 Dr. Richard Channick, dives into the evolving world within pulmonary hypertension -- interstitial lung disease (PH-ILD). He sheds light on why early diagnosis matters and how new therapies are transforming care. Learn about the latest FDA-approved treatment and what’s on the horizon. This Special Edition episode is sponsored by and . Hello, I'm Rich Channick. I'm Professor of medicine at UCLA Medical Center and Co-Director of the Pulmonary Vascular Disease Program at UCLA. I've been there for the last six-and-a-half years, but have been involved with pulmonary hypertension for over 30 years from my fellowship in the '80s onwards. So, I've gotten to see all the evolution and changes that have occurred in this exciting field.Today, I would like to talk about, really, an exciting, evolving area within pulmonary hypertension, which is pulmonary hypertension in the setting of interstitial lung disease or ILD, as we call it. The reason why this is exciting is that there actually are now emerging therapies for pulmonary hypertension due to ILD. The other important point to note is that this is actually more common than a lot of other forms of pulmonary hypertension. So, what are we talking about here? What's the terminology? Well, ILD means interstitial lung disease, and very simply, it's scarring or fibrosis within the lungs. When we talk to our patients who have pulmonary arterial hypertension, we often say, "Your lungs are fine. It's the blood vessels running through your lungs where the problem is." Well, in ILD-PH, that's not the case. In ILD-PH, the lungs are not normal. There are a number of things that can cause this fibrosis or scarring of the lungs.It can be due to some exposure. It can be due to another disease, like an autoimmune or connective tissue disease. It can be due to what we call idiopathic, or we don't know the cause for the pulmonary fibrosis. So, there's a lot of different causes. But what we do know is that in some patients, and maybe as many as 30% of patients, pulmonary hypertension or high blood-pressured lungs will develop as a result of the fibrosis.Again, that distinguishes it from other forms of pulmonary hypertension, like PAH, where the lungs may be fine, or left-sided heart disease or pulmonary emboli. This is a distinct subgroup within pulmonary hypertension, also referred to as group three. So, that's how we define the problem. What about treatment? This is, again, where the excitement is. Because there's now one FDA-approved therapy for ILD-PH, and then there are a number of others being looked at. There are some treatments for the ILD itself that are approved. Those treatments have variable effect, and there are some side effect issues.We know that if the pulmonary hypertension develops with ILD, that needs to be treated, because the prognosis in patients who have ILD-PH is worse than if they didn't have PH. The ability to treat the PH in ILD, and to show a benefit will help patients, certainly from a point of view of quality of life and their exercise capacity, hopefully will improve outcomes.Right now, in patients with ILD and ILD-PH, if it's severe enough, lung transplantation has historically been the only option, and that has its own problem. So, if one can find a treatment that can prevent or at least delay the need for lung transplantation, that's a big victory.In addition to the FDA-approved therapy, which is inhaled treprostinil, it's a long-acting prostacyclin-type drug that's also used in PAH. But the inhaled form was shown to be beneficial in ILD-PH, and therefore it was FDA-approved for that. In addition, there are a number of other drugs that are going through the development, mainly in ; similarly to inhaled treprostinil, looking at the ability of these drugs to improve exercise capacity, improve the hemodynamics, and hopefully improve the outcomes.The interest in ILD-PH has actually always been there, but it's always been a little bit of a head-scratcher. Because early on, there were some studies showing that some of the drugs we thought were exciting in PAH didn't necessarily help patients with ILD-PH. A couple examples actually were harmful. So, I think that slowed things down a little bit.But in recent years, again, with some of these newer therapies, and maybe it has to do with the availability of inhaled therapies, because when you have a lung problem, like ILD, PH is an attraction to inhaling the treatment where it gets right to the lungs, and may not have effects outside the lungs. So that, I think, raised the interest even more.Again, with inhaled treprostinil showing a benefit, a lot of the other newer agents, or that have new molecules, became aware really that this is a big need for which there's not a lot of effective treatments. I guess that's a little in contrast, of course, to group one or PAH, where we now have essentially 15 approved therapies. So, certainly, again, I'm a physician, I'm not a business person. But from a strategic business point of view, looking at an area where there's only one approved treatment versus 15, you could see where that might lead.The question is, how big is the pot? The pot may be bigger than we think of these ILD-PH patients, because one of the challenges is the recognition that a patient may be diagnosed with ILD or lung disease by an ILD specialist, but nobody's thinking about pulmonary hypertension. We believe, and we have some data to suggest, that may be underdiagnosed. So, the pot may actually be quite large of patients with ILD and pulmonary hypertension.I think it's very important that patients obviously are their own advocates in everything, but certainly in this area. If a patient has interstitial lung disease or pulmonary fibrosis and is being managed by even a specialist in that, to at least raise the possibility that, could they also have pulmonary hypertension? And let their healthcare provider know that they're concerned about that.Certainly, in the setting where, let's say, somebody's getting worse, so your symptoms are worsening, you're getting a little more short of breath, but the doctors are saying, "Well, your lungs are stable," the way we follow interstitial lung disease is a little different. We're getting pulmonary function tests, we may be getting imaging or CT scans of the chest. That may show stability of the lung disease, but yet the patient is feeling worse. That could be a sign or clue that pulmonary hypertension is developing.Thank you very much for listening. My name is Dr. Richard Channick, and I'm aware that my patients are rare. Learn more about pulmonary hypertension trials at . Engage for a cure: #phaware Share your story: Like, Subscribe and Follow us: . #phawareMD #PHILD #PHOCUSstudy #PROSERAstudy /episode/index/show/phawarepodcast/id/35841445

Episode 519 - Richard Channick, MD 05/05/2025

Ioana Preston, MD - phaware® interview 518 04/29/2025

Ioana Preston, MD - phaware® interview 518 Dr. Ioana Preston explores the emerging field of pulmonary hypertension associated with interstitial lung disease (PH-ILD). She discusses the growing recognition of PH-ILD, especially after the introduction of inhaled treprostinil as a treatment, and highlights the importance of early screening and diagnosis. Dr. Preston also delves into the challenges of treating rare diseases, the evolution of research, and the hope for future therapies that could significantly improve patients' lives. This insightful conversation sheds light on a critical yet often overlooked aspect of pulmonary care, urging clinicians to stay vigilant and informed about PH-ILD's complexities. This Special Edition episode is sponsored by . I'm Ioana Preston. I'm a physician and the pulmonary hypertension expert. I'm the Pulmonary Hypertension Director at Lahey Hospital and Medical Center in Burlington, Massachusetts. Today, I would like to talk to you about a rare form of pulmonary hypertension -- pulmonary hypertension associated with interstitial lung disease or ILD. PH-ILD is typically a disease of an adult. There is a small population of children, but it’s kind of a different entity. It's typically an adult disease because mostly adults develop interstitial lung disease. If we look at PAH Group 1, there are more women than men with a Group 1 PAH. This ratio does not translate in PH-ILD, where the population is more mixed. A typical PH-ILD patient is adult or elderly. They could be men or women. It's much broader in presentation. It's not a very specific population that we need to look at. That's very important, because we have to keep our mind open to screen all patients who have ILD for pulmonary hypertension. Now, at the 7th World Symposium on Pulmonary Hypertension that just took place in summer of 2024 renamed this entity and called it ILD-PH. So, interstitial lung disease with PH associated. Now, we haven't talked about this entity for a long time until recently. Why haven't we talked about it? Because there were no therapies available. We were not looking for pulmonary hypertension development in patients with ILD such as pulmonary fibrosis. It was kind of in the back of our minds as clinicians. But because there were no therapies available, we couldn't have any impact on the well-being of our patients with ILD who may develop pulmonary hypertension. Now, recently, after many, many years and many, many trials that failed, couple of years ago, a Phase 3 clinical trial showed that inhaled treprostinil is efficacious in treating pulmonary hypertension associated with interstitial lung disease. At that point, the door was opened and we clinicians understood that we need to look for this development in patients with ILD. We need to recognize it early. We need to screen for the presence of pulmonary hypertension in ILD. We need to treat it if it is available. Moreover, I think what is also important is to help the community develop other therapies for PH-ILD, because the story just begins. It doesn't end here. So, developing other therapies by enrolling patients with PH-ILD in clinical trials, I think it's a very important aspect that we need to focus on in the next years. So, going back to PH-ILD, what it means is the development of high blood pressure in the lungs or pulmonary hypertension in patients who have underlying forms of, or different types of pulmonary fibrosis. Clinicians now need to focus and learn how to screen and identify this entity. This educational program that occurred at CHEST 2024 is focused on teaching physicians and clinicians and healthcare providers how often and what tests are required to look for pulmonary hypertension, how to diagnose it, how to understand the severity of the pulmonary hypertension and how to treat it. So, now we have a better understanding and a more clear plan of how to approach pulmonary hypertension in the setting of interstitial lung disease. Going back 30 years ago, when I was a young physician in training, at that time, we had only one treatment for pulmonary arterial hypertension, which is Group 1 PAH. We had only Flolan. At that time, what was happening is we would diagnose patients with PAH and then we would wait for the disease to become severe, and then we would start therapy, because it was an infusion therapy. But this first drug that was developed in PAH opened a huge opportunity in research and clinical practice in PAH. Now, we have 14 therapies. Now, fast-forward in the 2020s, the same is happening. The same story is being repeated with PH-ILD. Up until very recently, all the clinical trials have failed, so we had no therapy for PH-ILD. Now, we have one therapy, so the door has opened. I am very hopeful that we will have more and more therapies in the future, in the next years to come to offer our patients to better control their disease, to improve their symptoms, and hopefully to have an impact on their life. That's why I'm really hopeful that we will have more options for our patients in the future. So, now fast-forward medical history to the 2020s, for PH-ILD there is a wealth of clinical research in clinical trials that are looking at different drugs and compounds to add on to the current therapies that we have available. There's very interesting research. I'm very hopeful that in the very near future, we will have more opportunities to treat PH-ILD effectively and improve the breathing, the activity, and the lifestyle of patients with PH-ILD. The rare diseases are challenging in and out of itself. The more rare the disease, the less experience the clinician has. We are humans and we think of more common things more often as a cause of our patient's symptoms. We forget the rare conditions, lack of experience, the fact that we in general clinical practice, not in my clinic, but in general clinical practice, you don't tend to see many patients with pulmonary fibrosis or ILD. Therefore, it's difficult to remember the subsets of comorbidities or other conditions that an ILD patient can develop. I think the first step is the fact that there's a rare disease, it's way in the back of the clinician’s mind. That's why it's very important to increase awareness of rare diseases such as PH-ILD. It's very important to remind clinicians to not forget about this entity and also to provide patients with pulmonary fibrosis information about this possible occurrence during their lifetime. But I have to say, if you look at different reports, I remember one report from Virginia from Inova Fairfax. It’s a large lung transplant center, and there patients with ILD get lung transplants. So, there is a strict requirement to look for pulmonary hypertension at the beginning of lung transplant evaluation, and also to evaluate for pulmonary hypertension just before they have the transplant. This report showed that at the time of transplant, 84% of patients with ILD just before they go to the OR, have pulmonary hypertension. So, we are talking about pulmonary fibrosis and all sorts of interstitial lung diseases. They're not common, they're rare. However, as the disease progresses, the vast majority of these patients will develop pulmonary hypertension. So, we really have to be cognizant of this important occurrence. My name is Dr. Ioana Preston, and I'm aware that my patients are rare. Learn more about pulmonary hypertension trials at . Engage for a cure: #phaware Share your story: Like, Subscribe and Follow us: . #phawareMD #PHILD /episode/index/show/phawarepodcast/id/35535560

Episode 518 - Ioana Preston, MD 04/28/2025

Raj Parikh, MD, MPH - phaware® interview 517 04/22/2025

Raj Parikh, MD, MPH - phaware® interview 517 Dr. Raj Parikh from Hartford Hospital discusses the development of the PH-ILD Detection tool, a screening tool designed to help detect pulmonary hypertension (PH) in patients with interstitial lung disease (ILD) at an early stage. Early detection is critical, as there is often a significant delay in diagnosis of PH in ILD patients, leading to worsened outcomes. This Special Edition episode is sponsored by . My name is Raj Parikh. I'm the Director of the Pulmonary Hypertension Center at Hartford Hospital in Hartford, Connecticut. I did a lot of my training kind of all over. I went to medical school in New Orleans, Chicago, ended up in Boston, and then made my way back home to Connecticut where I grew up. I've been at Hartford Hospital for the last four years. I started the Hartford Hospital's Pulmonary Hypertension Program from scratch, and I've been building it up since then. I got involved in Pulmonary Hypertension really during my fellowship at Boston. I really wanted to get involved in the rare disease space of PH, where patients are far and few between, but the ones that are there, they have pretty significant morbidity, mortality, and a poor prognosis. There's limited treatment options and there's limited specialty care for these patients. I really wanted to come back home to my state of Connecticut and build up a program in sort of the area that I grew up in. Today, I want to talk to you about the PH‐ILD Detection tool that Hap Farber, MD and I have been working on for a couple years. Now, it's in the community. We're encouraging all interstitial lung disease, ILD specialists, general pulmonary docs who see ILD patients to really use this as a way to work on early detection of pulmonary hypertension in interstitial lung disease patients. The reason behind developing the PH‐ILD Detection tool was to really focus on early detection and early screening for pulmonary hypertension that's associated with ILD. The impetus behind that was the development of the first and only FDA approved therapy for PH-ILD, which is inhaled Treprostinil. That was FDA approved back in 2021 after the INCREASE clinical trial. The PH-ILD Detection tool, the real spirit behind it is patients with interstitial lung disease that develop pulmonary hypertension carry extremely poor prognosis. They need more oxygen. Their quality of life is worse. Their longevity of life and their mortality is worse, as well. These are things we know. We've studied the PH-ILD population for years and years. This is well-established stuff. The key now that we have a therapy that can A, improve the patient's walk distance and therefore their quality of life. B, keep them from having any sort of clinical worsening like ending up in the hospital or having more right heart failure. These are all findings based on the INCREASE trial. So the key really is to detect the pulmonary hypertension as soon as possible in ILD, not just screen for it once a year, or test for it when you meet the patient for the first time and then test for it again every five years. This is not like colon cancer screening or lung cancer screening. This is active assessment every time you see an ILD patient, you should be thinking, “Okay, do they now have signs of pulmonary hypertension? Should I now be concerned about pulmonary hypertension or should I not be?" There is no standard protocol or standard practice when it comes to screening and detecting for pulmonary hypertension in ILD patients. Back in 2022, Hap and I really developed eight parameters for PH-ILD detection. We base these parameters off of things that you should routinely already be using to assess a patient's interstitial lung disease. We're talking about 6 minute walk tests, physical exam, pulmonary function testing, assessing whether the ILD patient needs oxygen or not, blood tests, CAT scans, and their prior history and comorbidities. If we take these eight parameters and we assess, okay, which parameters have a higher weight compared to lower parameters that have maybe not as much of a weight in terms of predicting for pulmonary hypertension? We took these eight parameters and basically created a 12 score tool. The score can be anywhere from zero to 12. Four parameters have a weighted score of two points. Four parameters have a weighted score of one point, therefore equaling a max score of 12 points. Basically, depending on what you score from zero to 12, you are stratified into either low risk for pulmonary hypertension ILD, intermediate risk for PH-ILD, or high risk for PH-ILD. A score of three or below is low risk. A score of four to five is an intermediate risk. A score of six or higher is actually high risk for PH-ILD. What does that mean? What does it mean if you're low versus intermediate or high? Well, that should really dictate what are your next steps. If you're low risk, you should reassess the patient at your follow-up visit, whether that's at three months or six months or whatever. If you're intermediate risk, you should get a screening echocardiogram for that patient to make sure there's no signs or development of pulmonary hypertension or right ventricular disease on that echo. If you're high risk, that's a score of six or higher on the PH-ILD Detection tool, you should not only order an echocardiogram, but you should be calling your nearby your local pulmonary hypertension specialist or pulmonary hypertension center and say, "I have a patient with interstitial lung disease. I have a high suspicion for pulmonary hypertension, can you get them into your clinic? Can you get them in for a right heart catheterization for a definitive diagnosis?" When we developed this score, we not only first developed it internally retrospectively, but once it was initially published, we discussed it and presented it at an oral presentation at CHEST back in Nashville, so that was a couple of years back. We then actually had seven centers used the score prospectively, and we validated the score by doing a multi-center validation cohort study. That study actually upheld a very significant area under the curve, very significant sensitivity and specificity for a score of six or higher to really strongly predict pulmonary hypertension in interstitial lung disease patients. As a result of this, we've been working on promoting the use of this PH-ILD Detection tool with local ILD specialists, local general pulmonologists. We use this score not only to predict or increase suspicion for pulmonary hypertension, we've done some other data and we actually presented this information at CHEST, this past year at Boston, where the higher the score you have, the worse your right heart cath hemodynamics are, and therefore the worse your prognosis may be. Some of that is almost common sense, but it's nice to kind of see pen to paper and see that correlation, as well. We've actually been working on seeing if we can create an app for this score. Now, Hap and I are creating an abbreviated version of the PH-ILD Detection tool to take it instead of eight parameters with 12 points, can we make it three parameters? Can we simplify it even further and make it as simple as possible so that the community pulmonary doctor, the community ILD specialist -- all of them can really focus on how simple it can be to detect and screen for pulmonary hypertension. Mostly because we know that once patients develop PH with ILD, the prognosis is so poor that we want to really act fast. We want to get them diagnosed soon. If we can diagnose them quickly, we can treat them with inhaled Treprostinil. We can mirror some of those outcomes that we saw with the INCREASE clinical trial, and get these patients having a better quality of life, better walk distance, lower proBNP scores, keep them out of the hospital, so on and so forth. We know from PAH pulmonary arterial hypertension and the REVEAL registry, there's almost a three year delay in diagnosis from the time a patient develops cardiopulmonary symptoms to the point B, which is when they actually have a formal PAH diagnosis. Three years. Those three years are met with seeing different specialists, being in and out of the hospital, having a worsening quality of life because they’re always short of breath and overall just poor more prognosis. If we take that, do we have that exact number when it comes to PH-ILD? No, but we do have an extremely wide range of epidemiology when it comes to PH existence with ILD. PH can range anywhere from 12% to 86% in ILD patients. Why do you have such a wide range? That's because we don't know when to look. We don't know how to look. A lot of times we don't even look and we're getting a lot of that data that 86% is sometimes showing up on autopsy results and transplanted patients. Then, we're looking back at those autopsy or transplanted lungs and saying, "Oh wow, those patients not only had ILD, they actually had PH as well." That's not something to necessarily be proud or happy about. So it kind of is in the same boat as, what is our delay in diagnosis? What is the window of opportunity that we need to really diagnose and intervene so we can make a difference? There will be a point of no return when it comes to ILD patients with PH. The same way there is sometimes a point of no return with PAH where the patients get so advanced that they're meeting them in the intensive care unit and they're meeting them really on what we call rocket fuel. These patients are looking for the Hail Mary to get better. You're not meeting them in the office, you're not trying to do a 6 minute walk test on them these patients can't even do a 6 minute walk test when they're sometimes at that point of no return. So really what is the sweet spot? I think it is trying to mirror what we went through with PAH several years ago. Developing a registry, understanding more of the referral patterns for PAH and we're trying to extrapolate all of that information and bring it now into this PH-ILD space, as well. I know I'm speaking to a lot of the providers and doctors who are involved with taking care of ILD patients, but really switching gears and thinking about it from the patient perspective. If you're a patient who has been diagnosed with any of the many subtypes of interstitial lung disease, I think it's important to be aware of this concomitant process that can occur and that can develop, which is pulmonary hypertension. It is difficult to tease out when you have worsening cardiopulmonary symptoms, worsening breathing symptoms. Is that the interstitial lung disease? Or is that something new such as pulmonary hypertension? I think it's important to think about that and be open with your provider, with your specialist, with your pulmonologist and say, "I'm now feeling this way, and I'm now using this much more oxygen, and wasn't my 6 minute walk distance this far last time and now it's down to this time? Could this be something else? Is this just not necessarily worsening interstitial lung disease or is this a second process?" A second process that now we know we can treat and we can make better. I think that's important to sort of think about. You can be your best advocate when it comes to going into your provider's office and really telling them exactly how you're feeling, and what you're feeling, and how it's different from prior. My name is Dr. Raj Parikh, and I'm aware that my patients are rare. Learn more about pulmonary hypertension trials at . Engage for a cure: #phaware Share your story: Like, Subscribe and Follow us: . #phawareMD #PHILD /episode/index/show/phawarepodcast/id/35840550

Episode 517 - Raj Parikh, MD, MPH 04/21/2025

Ron Zolty, MD - phaware® interview 516 04/15/2025

Ron Zolty, MD - phaware® interview 516 Dr. Ron Zolty delves into groundbreaking advancements in treating pulmonary arterial hypertension. From the historical use of vasodilators to the revolutionary promise of sotatercept and seralutinib, Dr. Zolty unpacks how science is moving closer to halting—and possibly even reversing—PAH. This Special Edition episode is sponsored by . My name is Ron Zolty. I'm a cardiologist working in advanced heart failure and heart transplantation. In my training, I had to do a lot of catheterizations in patients with pulmonary arterial hypertension. I trained at the University of Colorado in Denver, which is considered one of the best centers in pulmonary arterial hypertension. Working with patients in the cath lab, I fell in love with PAH, now leading a pulmonary hypertension program here in Omaha, Nebraska. Today, I would like to speak about some innovations in the field of pulmonary hypertension. As you may know, pulmonary arterial hypertension is a progressive, lethal disease. Patients, unfortunately, die from pulmonary hypertension if not treated. We should go back a little bit to the pathogenesis at the mechanism of what we believe causes pulmonary arterial hypertension. We know that when patients suffer from pulmonary arterial hypertension, they have what we call endothelial disruption. Endothelial is the inner layer covering the vessel of any vessel and especially the pulmonary arterial tree. If you have any damage of this endothelial layer, you are going to make some small thrombi, some small clots. One of the initial treatments of pulmonary hypertension was in the eighties to treat these patients with anticoagulation. Unfortunately, there was never any prospective study looking at the benefit of anticoagulation, only retrospective studies. Most of them showed that there is some benefit of anticoagulation blood thinner. However, because there was never any prospective study, it's just a 2b indication, not everyone needs to be on anticoagulation because again, it hasn't been shown really to improve survival in patients with PAH. The second main phenomenon that patients have with pulmonary hypertension is vasoconstriction. Vasoconstriction, meaning that the vessels tighten when you do have the disease. That's the reason for the three classic pathways we treat today. Pulmonary arterial hypertension, we know that patients with PAH have too much endothelin, which is one of the most potent vasoconstrictors we have in our body. So we block this endothelin causing less vasoconstriction and vasodilator effect. We also know that patients with PAH don't have enough prostacyclin, so we do substitute this lack of prostacyclin with some prostacyclin. We do have IV, inhaled, and oral prostacyclin to give to patients. The third pathway, which is also a vasodilator pathway, is the lack of nitric oxide. Patients with pulmonary hypertension have a lack of nitric oxide, and therefore we give them a PDE5 inhibitor like sildenafil or tadalafil or a SGC stimulator like riociguat to increase the nitric oxide pathway, increasing cyclic GMP. Cyclic GMP, really vasodilates. But all these therapies, these classic therapies do not stop the progression of the disease. They slow down the progression of the disease without stopping the disease. We also know, and that's the last point and the newest point I want to make, when patients do have pulmonary arterial hypertension, the wall of the vessel gets thicker. If the wall gets thicker, the lumen where the blood travels gets smaller and smaller. That's the reason why patients get less blood, less oxygen, less hemoglobin, less oxygen, more shortness of breath. This thickening of the wall gets what we call an increase in the resistance, leading to right side heart failure. About 15 years ago, there was a paradigm shift in the understanding of pulmonary arterial hypertension thinking, why do these patients have a thickening of their wall? It's because there is proliferation of endothelial cells, smooth muscle cells like neoplasia, like a tumor. Maybe if we block this progression, this proliferation, we may stop the disease and maybe even regress the disease in making the wall thickness less thick and smaller. Therefore, about 15 years ago, there was a very interesting clinical trial with a drug called Imatinib or Gleevec. Gleevec has, for patients with chronic myelocytic leukemia has been a revolution. Patients with CML in the past needed to have a bone marrow transplant. With the administration today of Gleevec it’s been a revolution and patients can live with CML, with this chronic myelocytic leukemia for years and years with absolutely no progression of their disease. Similarly to CML, the idea was maybe if we give an agent like Gleevec or Imatinib to these patients, we may stop the proliferation of endothelial cell and smooth muscle cells in the wall of the vessel and stop or even regress the disease. This trial, called IMPRES, was done about 13 years ago. The results of the study were absolutely beneficial. However, more than 30% of patients quit the study because of side effects, and also patients on anticoagulation, on blood thinners developed some intracranial hemorrhage. The FDA, because of the side effect, decided not to approve this drug for pulmonary hypertension. Just to give you a background that there is really a paradigm shift in the understanding of the disease. The new drug that has recently been approved by the FDA is being called sotatercept. That's another mechanism. We know that in our system, every cell has like a yin and a yang. There is activin, a system which stimulates the gene while the bone morphogenetic protein system is slowed down. The activin slowed down the proliferation of cells. In our system, in a normal system, you do have a balance between BMP and activin. We also know in patients with pulmonary hypertension, they do have too much activin. This new medicine, sotatercept blocks the activin. Activin is like an accelerator. It does promote proliferation of cells on the gene. If you block the activin, you recalibrate the BMP and the activin, reducing this proliferation of cells. Sotatercept was shown really to improve patient pressure as well as improving their six-minute walk distance as well as their quality of life. Sotatercept has been approved by the FDA for the last year and really has changed the field of PAH. Another group similar to the Gleevec, but as I said, Gleevec was not approved by the FDA because of the side effects. The idea was maybe if we could give a Gleevec as an inhaler without systemic effect, patients would absorb the drug into the lungs without having any side effects. There was a clinical trial with inhaled imatinib, inhaled Gleevec if you want. Unfortunately, the study was not positive, because most likely Imatinib was not absorbed by the lungs. However, another company was able to produce a super imatinib, which is being absorbed by the lungs called GB002 or showing a significant improvement in the pressure, as well as the PVR. Currently, there is a with this drug as an inhaler. So, in summary, I just want to say the drugs we have currently until now were vasodilators, they do slow down the progression of the disease but don't stop the disease. Now, we do have one drug approved by the FDA. Hopefully, a second one will be approved. These two drugs stop the proliferation that occur in pulmonary hypertension and hopefully will stop the disease and potentially even regress the disease. We are getting closer to maybe a cure of the disease with agents like sotatercept and . Thank you for listening. My name is Ron Zolty and I'm aware that my patients are rare. Learn more about pulmonary hypertension trials at . Engage for a cure: #phaware Share your story: Like, Subscribe and Follow us: . #phawareMD #clinicaltrial #PROSERA /episode/index/show/phawarepodcast/id/35842570

Episode 516 - Ron Zolty, MD 04/14/2025

Rajan Saggar, MD - phaware® interview 515 04/08/2025

Rajan Saggar, MD - phaware® interview 515 For decades, researchers have sought better treatments for pulmonary hypertension in interstitial lung disease, a condition that drastically impacts patients’ quality of life. In this episode, Dr. Rajan Saggar dives into the latest advancements, including new inhaled medications, upcoming clinical trials, and the push for more personalized treatment approaches. This Special Edition episode is sponsored by and . I am Dr. Rajan Saggar. I'm Co-Director of the Pulmonary Hypertension Program at UCLA, and my Co-Director is Dr. Richard Channick. We work at that program together. One of my main interests, actually, is pulmonary hypertension, complicating interstitial lung disease. Sometimes, interstitial lung disease can progress and become pulmonary fibrosis. So, along that spectrum of disease, pulmonary hypertension, which is Group 3. There's basically five types of pulmonary hypertension. There's five different groups. The one related to lung disease ... and again, that lung disease could be COPD or emphysema, or it could be interstitial lung disease - pulmonary fibrosis. Any type of lung disease is considered Group III pulmonary hypertension. The focus that we have right now is specifically with patients who have interstitial lung disease or ILD and pulmonary fibrosis. This is a field that I've been interested in for a couple of decades. There's been a lot of failed studies over the years. There's been a lot of attempts to use drugs that have clearly worked and are now FDA approved for the much rarer form of Group 1 pulmonary hypertension, which is also called pulmonary arterial hypertension. Those drugs, which are now FDA approved and thriving and doing well have been used or tried to be used in the Group 3 pulmonary hypertension setting. Unfortunately, the majority of them have failed. But the good news is recently, as of April of 2021, there is a medication that now is FDA approved, specifically pulmonary hypertension in the setting of interstitial lung disease or what we call PH-ILD. That is inhaled treprostinil. It's an inhaled medication that's taken four times a day. What's exciting now is that that started off a chain of events, which I think re-lit interest in this type of pulmonary hypertension. Now, there's several pharmaceutical companies that have shown interest, and more than that, have actually started clinical trials looking at the potential for other inhaled therapies. I say inhaled, because most of the therapies being looked at are inhaled therapies. The rationale there being you have a lung disease, specifically affecting the lung and you happen to have associated pulmonary hypertension. The idea being that if you inhale something as opposed to taking something orally or something through the vein, you might have more of an effect. You're delivering the drug to the area of interest. There's a lot of people and rationale for doing that and maybe sparing patients from the side effects of these drugs, when you inhale it, as opposed to giving it what we call systemically, either orally or through the vein. There's a lot of interest in the inhaled approach. Another product being looked at, and the reason I'm bringing it up is it's maybe close to being available and being launched, is a drug called Yutrepia. Again, another formulation of inhaled treprostinil made by a company called . We are recording this before the potential for launch, but it's possible that this drug could be launched as early as May of 2025. Again, the potential benefits of that drug, because one could ask, "Well, what's the point? You already have an approved medication that's inhaled treprostinil, as well." The touted benefits of that drug may be a decrease in side effects, particularly cough, which may allow more up-titration of that particular medication in terms of actual absolute dosing. What we do know is that more is better of this medication, inhaled treprostinil, in the setting of PH-ILD. If we can get to higher doses without having side effects, that would be the ultimate goal. Then, even a further goal would be to do that with, let's say, once a day dosing, for instance, to make the frequency less. That would be pie in the sky. But the point is there's motion here and there's movement, so we look forward to that. There's another formulation of inhaled treprostinil being looked at, which is by a company called Insmed, and they're also looking at a once-a-day formulation of inhaled treprostinil. Again, specifically in pulmonary hypertention with interstitial lung disease. That's not as far along as the Liquidia product, but that's also exciting, because again, you would be going to once a day, which would be a game-changer for a lot of patients. There's other companies looking at other formulations of inhaled treprostinil and trying to make that better, if you will. The reason I say that is because the current approved medication, inhaled treprostinil, is a drug which is four times a day and also can have cough associated with it, which is not atypical for an inhaled medication, but a lot of these patients have cough even from their interstitial lung disease to begin with. So, when we introduce a medication which sometimes exacerbates their pre-existing cough, it can become quite difficult. At four times a day, it can be a little cumbersome. There are other companies trying to improve the side effect profile perhaps, but also the frequency of how often they take it, trying to build on what's already out there. In addition to reformulating the treprostinil product there, there's also other companies looking at other mechanisms, just brand-new mechanisms. For instance, there's a drug called , which is made by . This is a drug also inhaled, a dry-powdered inhaler, which blocks certain molecules in the body called tyrosine kinases. It blocks several of these and has a lot of promise in PAH, so that's Group 1 pulmonary hypertension, but they've extrapolated that and now are looking at it actively in Group 3 pulmonary hypertension, specifically this PH-ILD, pulmonary hypertension with interstitial lung disease. That's exciting. The other medication that's being looked at is a drug made by , which is called , which is reminiscent of another pulmonary hypertension medication that we have in Group 1 disease, which is called Riociguat. But this is specifically being looked at, much like the Seralutinib product is being looked at in both Group 1 pulmonary hypertension as well as Group 3 pulmonary hypertension. Again, specifically pulmonary hypertension with interstitial lung disease. So again, you can see that the people are making newer medications not just for the Group 3 necessarily, but they're looking at it in Group 1, the more traditional pulmonary hypertension, the pulmonary arterial hypertension, but also looking at it in Group 3 pulmonary hypertension. That product with Pulmovant is also an inhaled medication. There is a protocol in motion, as we speak, looking at PH-ILD, which is exciting. It's a guanylate cyclase activator. It works in the nitric oxide pathway, which has been an old pathway used in pulmonary hypertension. It has some interesting benefits that are touted in terms of how it works in that pathway specifically. But without getting into the details, it is an old mechanism, but looking at it with new parts, if you will. That's Pulmovant. Again, that's their new medication, Mosliciguat. That's out there, as well. There's some other ones in the pipeline that are not as far along. There is a aldehyde dehydrogenase stimulator, which is being looked at. Again, very early. There's a drug that blocks hyaluronan synthesis, which is a drug that's coming out of Stanford actually, which is also very early in the mix. Nothing really ready for patients to engage in just yet. But again, the pipeline's pretty deep. It's very exciting because pulmonary hypertension with interstitial lung disease has really been a type of pulmonary hypertension yearning for a drug that we can actually give patients and actually get some relief. It turns out that this particular type of pulmonary hypertension, it causes a lot of illness, much more than the other types of pulmonary hypertension. These patients are unfortunately dealing with pretty much two lung diseases, they have pulmonary hypertension and they have this interstitial lung disease, so they tend to need oxygen. They tend to be very functionally limited. As anyone knows, living with oxygen in the house is hard enough. Leaving the house with oxygen, it becomes extremely difficult. Unfortunately, us in the respiratory world and the pulmonary world, we haven't really figured out how to best give oxygen outside of the house just from a comfort standpoint, and just making it easy for patients. It's still, I think, relatively cumbersome. I think that's another area that we should be focusing on to make living with oxygen easier, particularly outside of the home. So yeah, I'm happy to report that there's a lot going on in this field. From a clinical trial standpoint in the PH-ILD space, luckily so far, knock on wood, there's been sort of minimal overlap in terms of the trials being available. In other words, we don't have two trials or more going on at the same time for PH-ILD. For instance, I mentioned the Liquidia product, Yutrepia is farther along, so their study is finishing while the Pulmovant study with Mosliciguat is ramping up. So luckily, we haven't had the problem of having to try to figure out which study to go into, or which one to consider, or having choices in that matter. That really hasn't happened just yet, but I suspect it will, given the pipeline we just discussed. At that point, I think it's just an individual patient discussion, one-on-one discussion with the pros and cons, and probably has a lot to do with the side effects they're dealing with at some point, or inclusion/exclusion criteria. I think it gets into some of the details that we'd have to have an individual discussion with that particular patient to make the best decision with them. From a physician standpoint and trying to understand disease, any disease, not just pulmonary hypertension with interstitial lung disease, once we get more and more into it, we often start realizing that things can't just be lumped. Patients can't just be lumped into one category that you have PH-ILD and everyone will respond to drug X or drug Y. We all know that not everyone responds to available therapies. Or if there is response, there's a spectrum of response. Some are no response, some have mild response, some have significant response. But what makes this even more, I guess in some ways, difficult, but also exciting from a medical standpoint is that we are learning that there is an extreme amount of heterogeneity within PH-ILD. By that, I mean there's so many different causes and etiologies of ILD, and each of those patients with ILD has a different natural history of that specific ILD in terms of how it progresses. Some people are much more stable than others. Some people are progressively getting worse faster than others, and it has a lot to do with the type of ILD. Then, when you throw the pulmonary hypertension on top, the pulmonary hypertension itself has a heterogeneity to it in terms of how it progresses. Does it stay stable or does it tend to progress? Then, you can understand that when you're dealing with two diseases which are heterogeneous in their natural history, then it becomes a little bit more difficult to understand in terms of lumping people in one category. That's important, because the physicians in this space, and I being one of them, one of our tasks is to make sure that we understand what we call phenotypes. A patient's phenotype is not always the same with PH-ILD. Phenotype means the set of characteristics specific to that patient. There'll be patients just like that patient that fit into a certain phenotype. There's going to be multiple phenotypes in this PH-ILD space. We're learning about those. The reason it's important is because not only do they have different rates of progression or changes in their ILD and PH, but it also predicts who's going to need a lung transplant sooner perhaps, or who may be more responsive to a medication. Perhaps a patient who has idiopathic type of disease responds better to something than someone who has a connective tissue disease or vice versa. Those are the things that we need to learn about, so then we can inform patients better. Sometimes, that might trigger us off into saying, "Hey, you probably should think about this clinical trial, because the data with your type of phenotype may not be as predictable or as wonderful as we want." That's just the reality of the situation. Some people are going to have better responses than others. But that's important for us to identify that a priori so we can actually have a discussion with some real data so the patient can be informed, so we are not just making up stuff. We want to actually have real data, that hard data so we can actually help the patient make the best decision. I think that's really important. I'm Dr. Rajan Saggar, Co-Director of the Pulmonary Hypertension Program at UCLA in Los Angeles, California. And I'm aware that my patients are rare. Learn more about pulmonary hypertension trials at . Engage for a cure: #phaware Share your story: Like, Subscribe and Follow us: . #phawareMD #PHILD #PROSERAstudy #clinicaltrial #PHOCUSstudy /episode/index/show/phawarepodcast/id/35843325

Episode 515 - Rajan Saggar, MD 04/07/2025

Eric Austin, MD, MSCI - phaware® interview 514 04/01/2025

Eric Austin, MD, MSCI - phaware® interview 514 Eric Austin, MD, MSCI discusses early detection among people who are at risk of pulmonary arterial hypertension (PAH), particularly those who would have genetic susceptibility, or who may have congenital heart disease, or who may have connective tissue disease or other features that would make them more at risk of developing pulmonary arterial hypertension than the rest of the population. My name is Dr. Eric Austin. I'm a physician and physician scientist at Vanderbilt University Medical Center. I direct the Pediatric Pulmonary Hypertension Program at Vanderbilt and have done so since 2012. I have studied children and adults with pulmonary hypertension, in particular pulmonary arterial hypertension, but other forms as well, for nearly the last 20 years, with a particular interest in people who are at risk of pulmonary arterial hypertension for various reasons or are resilient to its development for reasons. I have great interest in the topic at hand today – early disease detection among people who are at risk of pulmonary arterial hypertension, particularly thinking about people who would have genetic susceptibility, or people who may have congenital heart disease, or people who may have connective tissue disease or other features that would make them more at risk of developing pulmonary arterial hypertension than the rest of the population. When you think about people who are at risk of developing pulmonary arterial hypertension, the most commonly thought about group in the current era are individuals who actually have familial disease; that is to say they have pulmonary arterial hypertension, or PAH, that occurs in families. This would be two or more family members with truly documented pulmonary arterial hypertension. We and others here at Vanderbilt and others around the world have been interested in studying families who are impacted by pulmonary arterial hypertension as a way to investigate the factors that lead to disease expression or presentation of PAH among people who are at risk. Years ago, in 2000, the initial genetic discovery was the association of bone morphogenetic protein receptor type II, or BMPR2 gene, with PAH. This association was discovered by several groups working in concert and several groups working independently, who discovered that single-gene mutations or rare variations in the BMPR2 gene can associate with pulmonary arterial hypertension. I intentionally said associate, because while I think they cause PAH, we don't actually definitively know that those mutations, ”cause pulmonary arterial hypertension.” We just know that beyond a shadow of a doubt, families with BMPR2 mutations are much more likely to have people with PH in them than the general population. Other genes have emerged over the past 20 years associated with pulmonary arterial hypertension, including mutations in the gene TBX4, mutations in ALK1 and endoglin, which actually also associate with hereditary hemorrhagic telangiectasia, SOX17, and several other genes. There's a smattering of genes that are now known to put a person at risk of developing pulmonary arterial hypertension. You may ask, "Well, what's the risk?" Well, that's the hard part. If you look at families who have individuals who have mutations but are healthy, it does appear that not everyone with a mutation in the genes that we think essentially “cause” pulmonary arterial hypertension will ever develop pulmonary arterial hypertension in their lifetime. Stated another way: a person with a mutation in one of the ‘PAH genes’ will not necessarily develop PAH. Most of this data actually is from BMPR2 mutation-carrying families, because mutations in this gene are by far the most common--about 75% of families that have PAH due to BMPR2 gene mutations. So most of our data emerges from those people. We know that on average, about 20% of all people who carry one of those mutations will develop PAH in their lifetime. But that's not a yearly risk; that's in their lifetime. We also know that mutation-associated PH can develop not only at any point in the life; but, it's actually different from men and women. People of female sex are much more likely to develop PH than those in male sex who have BMPR2 mutation. In fact, it's about a 42% lifetime risk for a female to develop PH if they have a BMPR2 mutation and about a 14-16% lifetime risk for men. For reasons we don't totally understand yet, although a lot of work has gone into this, as you can imagine, BMPR2 mutations don't equally cause PH between biologic females and males. That's a point of interest that makes you think, "Well, are the people of male sex resilient in developing PH, or are the females more susceptible, or somewhere in between?" It's probably somewhere in between, but we don't know for sure. You may then ask, "Well, what is the risk of a person to develop it in a given year if they have a mutation?" I told you there's a lifetime risk of about 42% for females and about 16% for males, but what is the yearly risk? Some of that work has now emerged from groups in Paris and in the Netherlands who have been studying individuals who have pulmonary arterial hypertension in families. Recent data from the French registry of these individuals suggests that about 2 to 3% of people per year in their cohort convert and develop PAH every year. If you take a group of people and they say to you, "Well, what's my likelihood of developing PH in the next year," it's probably about 2% to 3%. We don't know specifically yet. That's the best indicator we have. Here at Vanderbilt, we follow about 250 families who have pulmonary arterial hypertension in their family, and many of those have a BMPR2 mutation, and that's probably about the data that we have as well, though we have not published that. We also are now excited to be studying individuals who have BMPR2 or other mutation-associated PAH and then their family members who have those mutations but are healthy. We call that the RARE study, R-A-R-E. It's funded by the National Institutes of Health. I'm pleased to work on this with my collaborators and friends, Drs. Anna Hemnes and Evan Brittain, where we're studying people who have PH in the family, looking at healthy and unaffected as well as PH-affected people. We're interested not just in what makes the person who has the disease develop the disease, we're interested in what helps the person who is healthy stay healthy. By studying genomic and transcriptomic information, which tells us about their DNA and RNA, and then protein-based factors in their blood that may ultimately associate with whether they develop the disease or whether they stay healthy, we hope to learn more about ‘resilience’ to and ‘susceptibility’ to PAH. We hope that one day if we can figure out what factors are more present in the healthy people who should be sick and are less present in the diseased people who should not be sick, we can harness that to both prevent disease, but also maybe someday develop treatments built around that. You can imagine if there's something that... We find a protein that really helps someone stay resilient to or prevents them from developing PH even though they ought to have it, what if we could harness that as a therapy in the people who already have the disease? We're really interested in this notion, both here at Vanderbilt and around the world, of using people who have genetic susceptibility to understand the disease better and to understand how to prevent it and how to treat it better. You see a great example of this in the recent approval of sotatercept in the EU and in North America by the FDA. The drug sotatercept really emerged because of the genetic discoveries around BMPR2 and the associated pathways that go with it. We think that there's a lot more opportunity to learn a lot more from our genetically susceptible and genetically resilient people. There are also other forms of disease that associate with PAH. We know that individuals who have an illness known as HHT, or hereditary hemorrhagic telangiectasia, we know those people also are at higher risk of developing PAH in their lifetime. It's still quite rare for those people. It's not a common presenting symptom, but all of our PH clinics have multiple people with PAH and HHT, so it's common enough that we see at a fair amount. There are actually two different genes that most strongly associate with HHT, and that's endoglin and ALK1, or ACVRL1, and a few others. You probably don't care the names about them per se, but just to know that there are families of individuals who have HHT that courses through the family, and those people are also at risk of PAH. For people who have genetic susceptibility, whether it's HHT families or families with BMPR2 or other gene mutations, there's a little bit of controversy onto how often to screen the healthy people for pulmonary arterial hypertension. You may ask me, "Well, what do you do?" Most people would recommend that first-degree relatives, that is a mom, a dad, or a son or daughter, or a sibling, of a PAH patient due to a mutation like BMPR2 or HHT PH be screened every one to three years with a clinical evaluation and an echocardiogram for pulmonary arterial hypertension. I told you earlier, I direct the Pediatric PAH Program here at Vanderbilt, and here at Vanderbilt we actually tend to see the children who are at risk about every year with a clinical exam and an echocardiogram. I also like to get spirometry with DLCO to look at their diffusion capacity. If they seem at all impaired, we'll do a full six-minute walk test and lung volumes as well. But what we're doing is we're trying to screen them and perhaps detect PH earlier. If their echocardiogram is abnormal, you can imagine we would activate a full PH workup, including ultimately cardiac catheterization, because we think that earlier therapy will probably lead to better outcomes. We don't actually have that definitively proven, though, in clinical studies. But we also know that every year we have people who have syncopal events or pass-out events or other dangerous events that occur with their pulmonary arterial hypertension. I would be devastated to know that someone was at risk and we never looked for PH and then they died or had a bad event in the community because they passed out. So we screen our people who are children every year. In the adult world, you may see it more commonly every one to three years of screening those at risk. Another form of at-risk population would be the individuals who have connective tissue disease. The most common connective tissue disease that we know associates with pulmonary arterial hypertension is scleroderma or systemic sclerosis. But you can actually see PH in other forms, including lupus, mixed connective tissue disease and the like. So individuals, and they're usually but not always adults, who have connective tissue disease but are not known to have PH should be screened for pulmonary arterial hypertension every one to three years as well with a clinical evaluation and echocardiogram. Now, the reality is if you have connective tissue disease and you're well-connected, you're probably seeing your provider every three to six months, and part of the evaluation at every visit is considering progression of disease; is it possible the person has a new diagnosis of PAH? It's not extremely common in those groups to develop PH, but it certainly can happen. They can also develop pulmonary hypertension due to what we call Group III PH, which would be more lung-disease-driven, perhaps due to interstitial fibrotic changes in their lungs that can cause low oxygen tension and can cause PH, as well. So it's not always a pure pulmonary arterial hypertension Group I phenomenon. That's a quick once-over of the main types. The other things you see in children but not as much in adults, although you certainly can, is congenital-heart-disease-associated pulmonary arterial hypertension. So people who maybe had profound severe congenital heart disease that was operated on when they were young, such as a tetralogy of Fallot, or people who have less severe congenital heart disease, such as an atrial septal defect or a ventricular septal defect that's being monitored or was repaired, can all develop PAH at any point in their lifetime, as well. There's not strict guidelines for pulmonary arterial hypertension or pulmonary hypertension screening for repaired individuals who have had congenital heart disease in the past, but certainly if you were a provider and you saw a person with a history of congenital heart disease repaired that presents with unexplained shortness of breath or difficulty in breathing or other features that could be suggestive of pulmonary hypertension, then you would certainly want to activate some sort of more aggressive screening evaluation for pulmonary arterial hypertension for those people. Lastly, this is a group that may be less likely to seek care until they're very sick, or they may be depending on the scenario, would be people who are using recreational or other forms of drug use, such as amphetamines or cocaine. Those people are at higher risk of developing pulmonary arterial hypertension. So too are people who use the dietary drug fenfluramine, which has largely gone away in the United States and in Europe, but is known to associate with pulmonary arterial hypertension. This is not the diet drug that you're seeing now that's so popular across the world right now. Ozempic would be a common name for semaglutide and related GLP agonists. Those drugs do not, to our knowledge, associate with pulmonary arterial hypertension. But a traditional weight loss drug called fen-phen, or fenfluramine, did associate with pulmonary arterial hypertension. We still see it's used sometime among people with seizure disorders, by the way. I recently had a patient with this exposure, and we decided to stop that for safety reasons for that person. If you think about people who are at risk of developing pulmonary arterial hypertension, as I mentioned before it's pretty rare. Thankfully there's a lot more common diseases out there than pulmonary arterial hypertension. But it's really challenging to develop that disease because pulmonary arterial hypertension can significantly impact someone's quality of life and their survival over time, and so we like to be vigilant about it. If you ask the question, "How would one use the information we know about people at risk in a day-to-day basis," what I would say is that certainly for those individuals who are in a family known to have people with PAH in the family, two or more people in particular, of pulmonary arterial hypertension, it would be great for the family to be aware of it as a group and understand that, “Hey, cousin Jimmy died of pulmonary arterial hypertension and his son or his daughter also has it. I should think that if I ever feel short of breath, or if my daughter ever feels short of breath or has difficulty with walking or not really the same cardiovascular fitness that one would anticipate, that person should go be screened and seen by a medical professional.” One great way is to be aware of it on your own. It can be hard for families, because sometimes some of the people who are impacted in the family may be multiple branches of the pedigree or multiple branches of the tree away from the person who's actually at risk; that can happen. For individuals who have other risk profiles, like if they have connective tissue disease, if they have congenital heart disease, it's that partnership with your provider or your physician or your nurse practitioner or your PA or whoever you're working with for your care, and both of you together being aware that not only are you being cared for your current problems, but being vigilant for the associated conditions that can occur in the setting of your current diagnosis. If you have connective tissue disease, having a good discussion with your provider about, "Hey, Dr. so-and-so, thank you so much for your care of my rheumatoid arthritis, but what other things should I be thinking about as we move along through life? Do I need screening for pulmonary hypertension? Do I need screening for such and such?" Having that dialogue with your provider can be really helpful. Obviously, we're hopeful that would never actually come about and you would not be impacted by PH, but we would want to be on top of it in the event that you did have that happen. A thing that we're finally becoming a little more aware of in the PH community is the incorporation of people of all backgrounds, shapes, and sizes. For whatever reason, for many years, a lot of the individuals who we were studying at least, and even the people in our clinic, skewed toward a Caucasian population. But we've recognized in the last 10 years in particular that that needs to be rethought and more sensitively thought about. There are people in disadvantaged groups, individuals who are native people of the region, individuals who have African ancestry, individuals from LGBTQ+, who may not be seeking care or receiving care with the frequency with which other populations are. Many of us have worked with many groups to improve our distribution of information about pulmonary hypertension across all providers and into populations of individuals, as well, because that is a really important thing. I know Dr. Vinicio de Jesus Perez at Stanford has worked very closely with disadvantaged people across the world, which is another issue that's important. The PVRI (Pulmonary Vascular Research Institute) has been working on this ,as well, spreading the information about screening and about notification of PH and awareness both within North America, but also around the world in places that may be less able to access care or less able to access screening. Right here at home, I think we've done a much better job in the last 10 to 15 years of being aware of this and made sure that we are getting into all communities. Just like any other medical condition, if you don't have access to the care or you're not receiving care that's aware of the problem, you can miss it. I think through the years we've been not as aware of that as a group as we can be, and we've seen a real improvement of that over the last 10 years or so. I absolutely know that I have learned a lot about that, and so that's a really important point and it’s something that we, as providers, need to be thoughtful about. My name is Dr. Eric Austin, and I'm aware that my patients are rare. Learn more about pulmonary hypertension trials at . Follow us on social @phaware Engage for a cure: #phaware Share your story: #phawareMD /episode/index/show/phawarepodcast/id/34719895

Episode 514 - Eric Austin, MD, MSCI 03/31/2025

Kristin Highland, MD - phaware® interview 513 03/25/2025

Kristin Highland, MD - phaware® interview 513 Kristin Highland, MD, from the Cleveland Clinic discusses the importance of shared decision-making and patient preferences when treating patients with pulmonary hypertension. Dr. Highland, emphasizes that the patient is the true expert on their own body and that it is crucial to understand their perspective when making treatment decisions. She highlights the need to consider factors beyond just clinical outcomes, such as side effects, costs, and the patient's ability to manage the therapy. She underscores the evolving role of patient-reported outcomes in medical decision-making and the need for open communication between providers and patients to ensure individualized and preference-aligned care. I'm Kristin Highland. I'm a pulmonary critical care physician at the Cleveland Clinic in Cleveland, Ohio. I'm also trained in rheumatology. My focus has been rheumatic lung disease. In that space, I see a lot of scleroderma patients, and pulmonary hypertension is a frequent complication seen in scleroderma. I started at the Medical University of South Carolina and saw that we had a need for helping patients with pulmonary hypertension. At that point, back in the early 2000s, there was really only one therapy and it was a new therapy at that. I feel like I almost got into pulmonary hypertension on the ground floor. I started the pulmonary hypertension program in Charleston and then moved to Cleveland and have just been touched by the pulmonary hypertension community. It's a small community. You get to really know your patients well and their family members and other providers who have similar passion for this disease. Today, I'd like to talk to you about shared decision-making and patient preferences. When I think about taking care of a patient with pulmonary hypertension, I've come to realize that the patient is the true expert of their body. It is so important to meet the patient where they are, to understand their perspective when making decisions that are going to affect them. It's not uncommon that physicians and providers get caught up in the literature and which drug has the greatest survival benefit or is the gold standard treatment. We forget that there's always a cost to these therapies. There's side effects, expenses, there's pill burden, there may be self-image issues. There may be just the ability to execute the therapy, because you don't have good social support for some of these complicated therapies. I think there's a lot more that goes on in making these decisions. We have to make the right decision for the right patient. Sometimes, the enemy of good is better and sometimes we shoot for better and we make things worse. Decisions are trade-offs. I find it easier to think about these trade-offs when I think about cancer. Everybody can kind of think about the old-fashioned way of treating cancer where you get this horrible chemotherapy and your hair falls out and you barf your guts out. Would you do that for an extra week of life, an extra month of life, an extra year of life or an extra 20 years of life? Your tolerance of that type of treatment is going to vary based on what you get out of it. Would you do it if you're 99 years old versus if you are 25 and you have a two-year-old kid? Those decisions might influence what you're willing to tolerate and what you're not willing to tolerate. Patients are making these decisions all the time. They also are in the context of what else is going on. Often, pulmonary hypertension isn't their only problem. The average patient with pulmonary hypertension is on nine other medications. That's a lot to manage, a lot to deal with, a lot of side effects, potentially. I think it's important to really understand their perspectives, because I can prescribe medications all day long and if the patient doesn't want to take them for whatever reason, I'm not helping my patient. A medication cannot make you better if you don't take it. You have to find out what's going to work for that individual patient. I've also learned the hard way that patients don't read the textbook. They don't fit into these neat perfect diagnosis with zero other comorbidities. What works for one patient might not work for another patient, so treatment needs to be very individualized. I also think when we talk about severe diseases like pulmonary hypertension, there's no cure. People will eventually die from pulmonary hypertension and we have great therapies that are extending life, but we don't have a cure. I think as providers, we get so caught up in offering hope and offering our best therapies that we forget to address symptom management, exercise capacity and overall goals of care. Addressing the elephant in the room. Addressing that you're going to die from this disease, which is a really scary statement and is still an important one to have. We can have that conversation and say, I want you to have as many days on this earth as absolutely possible, and I want you to have the best quality of life with all of those days. But when you don't have a quality of life, what is your preference? And to meet the patient where they are, because that's a pretty individual decision, what the patient wants and what the patient doesn't want. It should not be my decision. It should not be my personal decision. I can say as I have gone through different stages of my life from being a college kid to a mother of a teenager, my decisions about my life have changed. Some patients want to do everything no matter what forever, and they are true fighters. I support those patients 100%. That's not the right answer for everybody. I think it's so important to meet the patient where they are and to remember that it's not just treating this disease with medications, but the goal is treating the disease with these medications, but with an emphasis on the outcomes that are important to the patient, which generally means giving them the quality of life that they want. I try to think about decisions that way. I think it's important to educate patients on what's out there. One of the things is that there has been a lot of great developments in pulmonary hypertension over the years. When I started, we had one therapy and now we're up to like 16 plus available therapies. Patients aren't dumb. They are on the internet. They are on this blog. They're members of PHA and other patient advocacy groups. They're wanting information. It is our job as providers to provide them that information and to provide it in a balanced way. When a drug is studied, generally, it's a very carefully curated population of study patients that are put in a clinical trial, which doesn't necessarily translate to all patients. We need to say this drug was studied in this way, in this particular population of pulmonary hypertension patients, and this is what we found. These are also the side effects that patients who were in the population experienced. Based on what I know about you, I think this might be a good drug to try, but maybe patient X has a certain comorbidity where I can say, because you have this other thing going on, it's possible that you don't tolerate this medication as well as we would like. So, we need to keep in contact. I need to know what you're going through when you take this medication, so I can either help you with side effects or we can decide that this medication is not a perfect fit for you. When we think about data that is on average the study of 1000 patients or the study of 200 patients or however many -- this drug improved hemodynamics or improved walk or improved time to clinical worsening. But that is a group of patients that this drug affected on average. There are patients in that one study that do great on that medication, and there's patients that do less great. There is a spectrum. I think it's important to say you're just one patient. You are an individual. These are things about you that I think might make this medication work or this recommendation of therapy, but here are things that we have to look out for. When I approach a patient, I always say, let's do what I call an N-of-1 trial, your own unique trial of this therapy. It sounds like a good idea. We're all on board. We're going to try this therapy and we're going to follow the results together to see if it's worth it. I learned this the hard way. My daughter has ADHD and I kept thinking her ADHD wasn't controlled, so I had just talked the pediatrician into increasing her dose and increasing it and increasing it. Well, she was hiding her ADHD medicine, because she didn't like the way they made her feel, which is why her ADHD wasn't controlled, which is why she needed escalating doses, because she never took the medication. I made that mistake with my own daughter. I could tell she was focused when she took her ADHD medicine, but she hated taking it, because it made her feel bad. Now, in pulmonary hypertension, we have 16 plus available therapies, clinical trials, all sorts of different things that we can try. It's really important to find the right combination of things that makes the patient feel better as opposed to what I'm reading about in a journal article. I try to think about that. In regards to clinical trials, it is so important for us to have clinical trials so that we can move the needle and the disease. This is how we're going to come up with a cure and we can't have clinical trials without patients. We do a lot of clinical trials at the Cleveland Clinic. I offer these trials as an option for patients. I make sure that they understand if you're participating in a clinical trial that is always voluntary. You can decide you want to do the trial today and tomorrow you can say, I don't want to do it anymore, and you can quit. Clinical trials are voluntary. Whether you participate or not should not affect your care with your healthcare provider. It certainly does not affect the care they get at the Cleveland Clinic, whether they participate or not. So that needs to be very clear. I also say, the reason it's a clinical trial is it’s an experiment. We do not know if it works. It might not work. It might make you feel worse. We have no idea. That's why it's a clinical trial. It needs to be studied. But then I say, I am so grateful for the patients who do participate in clinical research so that we now have more and more options for our patients with whatever disease, but participating in a clinical trial, that's an individual decision. Sometimes, it's just a pain in the neck to come in as often as you have to for clinical assessments in a trial. Some patients work and they just can't take that amount of time off work or they live too far and they don't feel well enough to travel that far, that frequently. Some people don't want to feel like they're being experimented on. That is 100% understandable. Other patients say, I don't know if this'll benefit me, but if I can help contribute to the knowledge about pulmonary hypertension, that's my way of giving back. A lot of patients want to participate in clinical trials for altruistic purposes. They just hope that someone else can benefit from the science. I'm certainly grateful and amazed by the number of patients that want to do that. Whether a patient participates in a clinical trial or not, again, I think is 100% their decision. I just put it out there as this is also an option. Just the same way I say, and so is participating in pulmonary rehabilitation. Shared decision making, I think has evolved over the last 20 years. The FDA, for instance, seems to have put more emphasis on what we call patient reported outcomes, how a patient functions and feels. Not just do they live longer, do they walk farther? Does their PVR drop? The FDA recognizes the importance of therapies. Not only do they want to have a biological improvement, but they need to have an improvement that the patient cares about. We're getting more and more data. Although there's some alignment between the providers and the patients, it's not complete. For instance, I've recently been on a project where we've looked at the provider recorded functional class of a patient. So WHO functional class is something that is recorded in pulmonary hypertension. Functional Class I patients don't have symptoms. Functional Class IV, they're more or less symptomatic at rest. Function Class II is they have some symptoms. Function Class III, they have more. Function Class IV, they're at rest. I was involved in a project where providers, which is the usual way of assessing functional class. But then, we had patients report their own functional class, assess it themselves, say, “Well, I think I'm a Functional Class II or I'm a Functional Class I. What we found was about 50% of the time the provider and the patient matched, but 50% of the time they didn't. The providers both over and underestimate functional class. The cool thing was that the patient reported functional class had better alignment with quality of life measures like emPHasis10, which is a pulmonary hypertension quality of life tool, and the SF-36, which is like a general health quality of life tool. What the patient says about their functional class aligns more strongly with these other measures than what the provider thinks about the patient's functional class. Although there is some alignment, it's not perfect. I think we're getting more and more data that patient perspectives matter. I also think patients have more ready access to information at their fingertips, because of the internet and blogs and websites. They are coming in educated, asking questions about various therapies. They want to know about them. They want to know if they're a candidate. They're hearing about other patients' experiences, because patients are blogging and saying, “Well, I tried this and this happened and it was great,” or “I tried this and this happened and it was horrible.” They're hearing all these stories. Patients certainly come in with a lot of questions. I think that's the challenge to make sure that we are spending enough time with our patients to answers those questions, to discuss the options, to provide them fair, balanced information. This is what the drug can do, but here's also the side effect profile of the drug. And to think about, you have to take this drug three times a day or four times a day or once a day, or it's a continuous infusion, what does that look like? Or you can take this drug and oh, by the way, your copay is humongous, because these drugs are expensive. It's going to be challenging to get prior authorization. There is a very, very unfortunate financial piece to these decisions. Also, I find as a provider, sometimes it's really hard to discuss. You're talking about therapies. You want to help your patient. You want them to live as long as possible and have the best quality of life as possible. It's hard to have a conversation around hope and then also have a conversation around goals of care and what are your thoughts about death and dying, or do you have a living will or palliative care, which I think are really important topics. Sometimes, these are topics that scare patients that they don't want to talk about, they're not ready to talk about, but sometimes they're topics that patients do want to talk about, but they don't know how to bring the question up. There's this elephant in the room that nobody's talking about. We're doing a disservice to our patients if we don't bring that part up of care. Often, patients hear the word palliative care, and it's scary. Our palliative care group prefers to be called quality of life doctors. Just because you have this disease doesn't mean we can't really work on addressing your symptoms the best way possible. Treating someone with pulmonary hypertension medications and also better managing their symptoms are not mutually exclusive. You can do both at the same time. I think providers have a hard time balancing those conversations, because you certainly want to leave the patient with hope. Always want to leave the patient with hope. But honesty and addressing the scary topics is super important too. I try to figure out where the patient is and how receptive they might be. Sometimes, these harder conversations have to build with time and patients have to be ready to have them, but they have to be given permission that the conversation can be had when they are ready. It's hard. It's hard to get through all of this in a short follow-up clinic visit. That's the challenge of taking care of the patient, but probably the most important part of taking care of the patient, in my opinion. I'm Kristin Highland, and I am aware that my patients are rare. Learn more about pulmonary hypertension trials at . Follow us on social @phaware Engage for a cure: #phaware Share your story: #phawareMD /episode/index/show/phawarepodcast/id/34697530

Episode 513 - Kristin Highland, MD 03/24/2025

Jimmy Ford, MD - phaware® interview 512 03/18/2025

Jimmy Ford, MD - phaware® interview 512 Jimmy Ford, MD discusses the 7th World Symposium on Pulmonary Hypertension and his work on the in PH Task Force. My name's Jimmy Ford. I work at the University of North Carolina at Chapel Hill where I direct the pulmonary hypertension program. I got into pulmonary hypertension when I was a pulmonary fellow about 17 years ago. At our institution, there was not really a formally organized program or cohort of patients being followed by a pulmonary hypertension specialist. Also at that time, there were a number of new therapeutics coming online and being developed, so it was a very exciting time. I managed to cohort together an increasing number of patients throughout my fellowship to the point that once I was done, they didn't want me to leave. I managed to stay on and continue to grow the program, and here we are today. I'd like to discuss really a new, important task force that was included in the World Symposium on Pulmonary Hypertension. At the previous event, in 2018 in Nice at the Sixth World Symposium, we had the first patient perspective task force. Then, back this past summer, in Barcelona, now the second . It's really, I'd like to think evolved quite a bit since the first one, and appropriately was positioned as the first task force presented at the World Symposium fittingly, since everything should be centered around the patient. It really helped to set the stage for a lot of important discussions about patient care, and brought to the forefront a number of new issues with regards to patient's perceptions of care, how they access care, how they are or are not included in important shared decision-making approaches, and making sure that their individual treatment goals are being addressed and met. I think it's a really important topic that we'll see increasing focus on as the years go by. I think as providers we tend to get tunnel vision sometimes about we have a disease and we must treat the disease, but all the while forgetting there's a patient who's dealing with that disease. I put a quote up by Sir William Osler when I presented our recommendations at the World Symposium, and he stated that a good physician treats the disease, a great physician treats the patient who has the disease. I think that's really the spirit of this. Every patient's journey and experience of pulmonary hypertension is different. Every patient's goals for therapy and what they want to achieve in terms of treatment are different. I think if we just take a sledgehammer approach of we must treat this disease, must treat this disease, and don't consider the individual patient's journey and how they're dealing with it or struggling to deal with it and what they want to achieve with therapy, then we're really doing them a disservice. I think that's why we're seeing this percolate ever more to the top of the agenda on the World Symposium and other important pulmonary hypertension meetings, as well. We'll really need to lean on patient associations and patient advocacy groups increasingly as time goes on to really get their input collectively, as well. They really serve as an important avenue for patients to collectively advocate for their voice to be heard, and also a mechanism for their voice to come through in cooperation with industry and provider professional societies. One area that we talked about was patient reported outcome measures, or PROMs, as we call them for short, which are tools that have been systematically developed to essentially capture patient's quality of life. There are a few different tools out there, some of which are specifically developed for pulmonary hypertension, some of them are more generic. At the end of the day, the intention is to capture not only symptoms of the disease, but also how the symptoms affect the patient's life, and also how the effects of treatment affect the patient's life. Trying to find ways to get clinicians to adopt a more systematic inclusion of the use of those tools in clinical practice is really something that we advocated for. Not really coming down to preferring any one particular instrument over the other, but simply just to try to encourage patients to do this systematically. There are a lot of potential benefits. It helps to facilitate the shared decision-making approach, in that you are collecting quality of life information from the patient in a structured manner that is repeated hopefully in serial encounters over time, such that you can compare and contrast how the patient may or may not be achieving a better quality of life with therapeutic interventions or having their treatment goals met. And really thinking about, well, how can we better facilitate their use in clinical practice? The one thing we always hear from providers is that there's just not enough time in the clinical encounter to do this, right? It's a tool you've got to give to the patient, they've got to fill it out, it takes some time. Ideas like electronic versions of these tools, which are in development, and can be provided to the patient ahead of a visit. Patient can fill them out, it doesn't take time away from the clinical encounter. We can set reminders to have the patient fill them out or to remind us to have the patient fill them out at certain intervals. They also can be tools which help to determine whether quality of care that's being delivered at a particular center is at a certain desired level, and hopefully improving over time, as well. I think as these are developed further and developed into formats that are more readily digestible and can be completed more easily in the clinical encounter or even in advance of it, we will see better adoption of them. Another issue is, thinking more globally, how many languages are some of these translated into? If you're looking at the pulmonary hypertension-specific PROMs, I think the most that any one of them is translated into is about 20 to 30 languages, whereas some of the more generic ones are available in well over 100 languages. We prefer to use a pulmonary hypertension-specific PROM, that was our recommendation. But we still have to do a lot more research about things like, how often should patients be filling these out? And do they potentially have some value in conjunction with the risk assessment tools to help actually inform the risk profile of the patient too, not just their quality of life? We do have a few studies out there that have shown that the PROM scores, particularly for EmPHasis-10 and the SF-36 tool ,at the time of diagnosis, have translated with outcomes over time. We still have a lot to learn and explore about how to best utilize these tools, but I think at minimum we need to find a way to start using them more routinely in clinical practice. One thing we did is we polled a variety of patients, providers, and patient advocates throughout various areas of the world across almost every continent. They were distributed all throughout South America, Africa, Europe, Oceania, Asia, so a really varied sample of patients in terms of social situations and geopolitical situations. There were a lot of similar recurring themes that we saw from these survey responses, including generally an impaired lack of access to expert pulmonary hypertension care in centers. In conjunction with that, relatively poor access to diagnostic procedures, particularly the most important one in pulmonary hypertension, the right heart catheterization. If we're looking at this globally and we can't get a significant number of patients to right heart catheterization, the discussion about the disease is moot because we don't even have a diagnostic foundation to begin with. We talked about some initiatives that have been employed in the past and that might be expanded upon to improve access to the right heart catheterization, whereby medical centers throughout Europe, South Africa, United States, have partnered with underserved areas, particularly two in Africa, Uganda, and the Morning Star Clinic in Zambia, where cardiology teams have actually traveled to these places and provided expertise, equipment and ability to get those sites set up to do right heart catheterizations. But again, that's just a few places, and there's still large swaths of the world where access to that remains poor. Similarly, distance that patients are traveling to pulmonary hypertension centers can be quite far either in time and or distance. Clearly, we need more expert centers, more expert providers throughout the world in underserved areas. It's interesting, even in the relatively underserved or global south areas of the world, even beyond those in fairly well-resourced nations within them, we see significant disparities in healthcare access and delivery. It's really a complex problem, which certainly is not limited to just pulmonary hypertension, but I think when you've got a serious chronic, relatively rare disease where the therapeutic regimens can be quite complex, these obstacles become all that much greater to overcome and, more importantly, to address. Another few areas that we addressed in our task force included, for example, climate change. In general, the idea of adverse humanitarian situations, which can include climate change, armed conflicts, pandemics, all of which we are seeing unfortunately throughout our world with increasing intensity and frequency. So recognizing what are some of the challenges that each of these adverse situations brings, particularly to a pulmonary hypertension patient. For example, with the topic of climate change, we know that poor air quality, increased air pollution, extreme heat and weather events leading to storms, flooding, et cetera, can really cause a lot of issues for patients with cardiopulmonary diseases, can increase symptoms of dyspnea, cause acute exacerbations. There's the potential for waterborne and vector borne illnesses with flooding, all of which on top of a complex serious disease like pulmonary hypertension can really spell a lot of trouble. Then, with regards to armed conflict, like any individual, and especially those dealing with chronic rare complex diseases like pulmonary hypertension, life is disrupted significantly, right? People are displaced potentially, their access to medications and care is interrupted substantially due to their likely geographic displacement or to the destruction of that healthcare infrastructure or medications. Really the best way that we could think of to try to address or mitigate some of these things is with advanced contingency planning of multiple different types, forming cross-border partnerships, really educating the patient about what to do in these situations, making sure that they are maintaining a supply of their medications, that they know where to go in the event that they cannot reach their usual care team, that they have access to telecommunication devices if telemedicine or e-health is necessary to be employed. Every situation is different, of course, with these adverse humanitarian situations, but we thought it was important to call them out as they're becoming increasingly problematic, and to try to come up with strategies to preemptively deal with them. Unfortunately, methamphetamine use is becoming an increasingly noted cause of pulmonary hypertension that we see in a lot of pulmonary hypertension clinics across the US and even the world depending on where you are specifically. We didn't address that specifically from the context of the patient perspective, but it certainly is an emerging problem, which does have some geographical predilection. In my part of the US, it's not so much of an issue. I know in many parts of California and Western US up to and including regions around the Mississippi River it is a huge problem, and probably other urban centers throughout the eastern US too. I think it really depends on where your practice is located. Meth use really plays into the idea of social determinants of health. The reasons that people get into the use of methamphetamine are likely related to a lot of what we call allostatic load metrics that affect one's health. Being poorly, financially resourced, having low general and or health literacy, and having a lot of life or psychosocial stressors which lead one down the path of substance abuse. It is relevant in that regard in terms of addressing the social determinants of health. So probably should be something that is addressed in the next patient perspective task force, particularly if it continues to be an ever-increasing problem in pulmonary hypertension clinics. One other important aspect is increasing the diversity of patients that we include in our registries and our clinical trials. For the longest time, those cohorts have consisted largely of non-Hispanic white patients, if you look at the percentage. It's not getting any better as time goes on from when we really started doing pulmonary hypertension trials a few decades ago, to even our most recent trials with Sotatercept, for example, we're still seeing overwhelmingly non-Hispanic whites being enrolled. That's problematic in the sense that we sort of take the findings and results of these trials as generalizable to our entire patient population that we treat in the real world outside of clinical research. That may not be true. As it is, consider that we already see significant variability in treatment responses to pulmonary hypertension therapeutics between individuals even within the same race. You can imagine that genetic pharmacogenomic differences certainly exist on a racial basis that we're not capturing in our clinical trials. I think we need to be much more intentional about enrolling certain percentages of races and ethnicities in clinical trials so that we can stand on a better and firmer ground when we say that we can truly generalize these findings across our clinic patients. This is really just a top-line look, a 30,000 foot view, if you will, of the world of the patient perspective and pulmonary hypertension. I think it's a really challenging area because so many factors inform it, right? We'll continue to uncover more, and we should continue to uncover more as time goes on. But I think it's important to really tackle the ones we've identified now head-on, and be ever mindful of emerging ones like methamphetamine, as I mentioned. My name is Dr. Jimmy Ford, and I'm aware that my patients are rare. Learn more about pulmonary hypertension trials at . Follow us on social @phaware Engage for a cure: #phaware Share your story: #phawareMD /episode/index/show/phawarepodcast/id/34683465

Episode 512 - Jimmy Ford, MD 03/17/2025

Hap Farber, MD - phaware® interview 511 03/11/2025

Hap Farber, MD - phaware® interview 511 Harrison "Hap" Farber, MD gives a recap of PHenomenal Hope 2024, emphasizing its patient-centered approach to pulmonary hypertension (PH) research. He highlights patient stories, particularly Eric Borstein's inspiring journey of recovery and advocacy, and underscores the importance of patient-provider collaboration. Additionally, he addresses challenges in PH care, such as inclusivity in clinical trials, the role of AI in medicine, and the complexities of treating patients with substance use disorders. I’m Hap Farber. I do pulmonary hypertension at TUFTS Medical Center in Boston. This is the second edition of . It’s different than other symposia in that it’s much more focused on patient needs, patient opinions, patient outcomes, and what we can do in the future to improve all of those rather than just basic science or clinical science that most PH symposia cover. For example, in this year, we had a patient come talk to us about what the journey’s like for a patient, which was just incredible. People just were picking their jaws up off the floor. It was just amazing. This patient, Eric Borstein, is like the perfect patient. He’s the perfect patient in part because he has the resources to be the perfect patient. Part of the problem is the bulk of our patients don’t have the resources to be the perfect patient. Eric’s had a remarkable response to drugs, thank God. Secondly, he’s incredibly articulate about the journey he’s gone through. Thirdly, for those of us who do this a lot, he’s like the perfect PH patient that we never have, but it then sets a bar for us and our patients to try and reach that bar. As he said in his talk, he was a typical male. He disregarded or didn’t pay any attention to his symptoms, which went on for multiple years before he actually almost died because he’s a typical guy. You know, “It’ll go away.” He ended up initially diagnosed at such a severity that he ended up in an ICU, almost died from right heart failure, ended up on systemic therapy and had a remarkable response to it such that over time his right ventricle got better, he got better, and he was able to transition off onto oral meds. In this whole time, he had come to the conclusion he was going to somehow beat this disease. Through his wife, they assembled basically a team. He’s got a sports psychologist, he’s got chefs, he’s got physical therapists, but he has the wherewithal to be able to do that. Then, he started walking. By walking, he could convince himself that he was getting better and that every step he took was another step towards beating this disease as he says. In addition, for him, it seemed like that walking mitigated the side effects from the drugs. He just kept walking farther and farther. The guy walks about 12 to 15 miles a day. He doesn’t just walk, he walks four miles an hour. I mean, he’s doing a mile in like 14, 15 minutes. I mean, he chugs right along there. There’s no facade here. He realizes that he was incredibly fortunate to have survived and to have the response he did. As he says when he talks, what he wants to do now is basically devote whatever time he thinks he has left to patient education, patient advocacy, stuff like that. I think in the past, one of the biggest problems is there’s a disconnect between patients and their providers. And a lot of the talks (at PHenomenal Hope 2024), showed you that there’s a disconnect. Not only showed you, but tried to investigate why there’s a disconnect. Part of going forward with PAH is in a way to make it a more perfect patient, you need the patient to understand why you do what you do, explain to them why you do what you do, and have them work with you as a team, which is something that we at TUFTS have done for years. You can’t treat the patient as a number. Despite the fact that in a lot of practices now because of the volumes or the constraints, they’re considered a covered life or a number or whatever you want to call, they’re not. You have to think about, if I were that patient, what would I want to know? How would I want to be treated, and what input should or would I have as to what happens to me? Unfortunately, in a lot of situations, that’s not done. None of the three are done. It works better when all three aspects are done because then you get to this issue that you’re working as a team. Patients understand more of why you’re doing what you’re doing, what you’re doing, what you expect to happen, how you’re going to manage their side effects from the drugs, et cetera, et cetera. As I said at the beginning today, when we did the first one last year, I was scared that nobody was going to show up. Then this year, I looked out there, and I was like, “Oh, my God, look at how many people have actually shown up.” We actually have to keep getting better and better and upping in our game. So far, at least the feedback that I’ve gotten over the course of the two symposia has been nothing but positive. Nobody said anything negative. Part of that is the fact that we touch on subjects that most PH symposiums don’t touch. We are dedicated to patients and the advocacy and all that stuff. It’s interesting now though, since we started doing it, like at the World Symposium this year, they actually had patient input for the first time ever in their 25 years of doing it. So this first time they’ve actually asked patients to give their opinions and stuff like that. I think a lot of it goes to the idea that despite what we believe or some of us believe, patients actually want to be part of this team and want to feel like their opinion and/or their thoughts of what happens to them is actually valued, which it should be. My nurse practitioner and my fellow could tell you we go through everything with the patients, and then we go, “So what do you think?” And see what they say. Then, I’ll go, “Well, it’s okay. We can do that. But you might want to expand that horizon a little.” That’s important. The patient wants to be included. No matter what docs think, patients don’t want to be talked to. They want to be included in the discussion. It’s not happening uniformly. Some of the same problems that we had 10 years ago, we still have. You have to realize that this symposium is also very different than others in that this is driven by the abstracts that are submitted, not by my want as to who, whatever. So depending on whoever’s writing the abstracts puts in, then we decide. The one topic which I put in there because I knew it would be above everybody but within five years clearly will not be was the discussion of bioinformatics and AI. Like it or not, that’s where not only the world, but the world of medicine is headed. You can’t stop it. It’s like a tsunami. Now, the question really is going to be, how do you harness it for good and not bad? There’s going to come a point where depending on how good these machines are, they’re going to know more than we do. You have to understand that as much as you may or may not like AI and bioinformatics and all this stuff, you’re not stopping it. There’s too much momentum and too much money involved in it. In simplistic terms, medicine has tried to simplify things. You have a disease, you think you have the cause or the etiology, and you make a drug to treat it. Well, PAH is not that simple. It’s multiple different pathways that probably lead to the same node that then from that node pathology develops. The question really is, what’s upstream from that node? Are there pathways, molecules, enzymes, whatever, that you could exploit either with currently available drugs that you reposition for this or new drugs that target these pathways? That’s what it’s trying to tell you. Or the other possibility is that there’s so much overlap that you never realize between this and disease Y, that you can take the things you’ve learned from disease Y and apply it to PAH. The closest analogies are, if you look now at the neoplastic drugs, the cancer drugs, those guys are 10, 15 years ahead of us easily. But they do have the advantage of they have tissue. They can sequence tissue. You get a chunk of tumor, you sequence it, you find out where the abnormalities are, then you make a drug. When you see all those ads on TV, that’s where those things came from. They’ve got an antibody that just blasts that mutation. That’s good because it’s more specific, it reduces side effects, it decreases off-target effects and stuff like that. We don’t have that yet, but we will if they keep going like this. So since we don’t have tissue, we have to make our own tissue by computational biology and with computers and AI and all this, figure out if we had this chunk of tissue, what it would look like. One of the areas that we focus on now on both years we’ve done this as unmet needs is the patient who does meth. The biggest problem with meth, aside from the fact that their pulmonary hypertension tends to be pretty severe by the time they’re actually diagnosed, is the fact that you not only have to deal with the pulmonary hypertension, but you have to deal with addiction and other social issues that come along with addiction and things like that. Unless you can navigate those waters at the same time, it’s hard. It’s especially hard in a way because in the U.S, not so much in Massachusetts, but in the U.S, the access to mental health care, it’s just pathetic. It’s been cut and cut and cut and cut such that to get somebody into a mental health program with some expediency is really difficult. It probably depends on where you are. It’s clear that out in the West, meth is a huge issue. However, there’s meth in New England, Western Mass, Northern Vermont, Northern New Hampshire, it’s there. Part of the problem is I think they’re used to looking for it. People in the Northeast are not so used to looking for it because we don’t believe that it’s here, but it is. We’re more used to looking for cocaine whereas in the West, they have almost no cocaine. Everybody’s doing meth. In the West, if you don’t think of meth and you don’t screen your patients for meth, or God forbid, you don’t even ask them about it, you’re never going to find it. In New England, for example, I think fewer physicians actually ask about meth or actually do tox screens for meth or stuff like that. We ask every patient, every new patient about meth. We do. It’s surprising how if you ask, you’re surprised. All of a sudden this person goes, and you’re like, “Whoa, okay.” I always use this example. I saw this woman with idiopathic PH, was sent down from New Hampshire, suburban housewife, two kids, picket fence, dog, station wagon. So we’re in there, and I go through my spiel and go, “So any history of meth?” She goes, “Yeah, I used to be a total meth head.” Her husband obviously knew nothing about this. He’s looking at her like, “Say, what?” I said to my nurse practitioner at the time, would you love to be a fly on the window on that car ride on the way home? Part of the issue that’s been raised not only in PAH but in other disease states about clinical trials, clinical trial designs, registries, are the fact that they’re not inclusive. That presents several problems. One is since drugs are approved based on clinical trials, if the clinical trials are not inclusive, you then don’t know if the drug works across the board or did you just happen to hit an ethnic population in whom it works? There are multiple reasons why clinical trials and stuff have not been inclusive. We went through a lot of them. Some of it has to do with physician bias. Some of it has to do with patient bias. Some of it has to do with the economics of clinical trials. A lot of patients who work can’t just take the day off and come in and do their clinical trial visit, even if you do buy them lunch or pay for their parking, because their boss doesn’t understand. They’re risking their job. Then, there are the people who have tremendous suspicion about the medical community based on previous clinical trials to say the least unethical. A lot of these all lead up to the fact that it makes it difficult in some places to recruit minorities into clinical trials so that you get those data that you actually need to know. It’s clear now that some of the pharma companies are actually trying to, in their trials, which they never did before, report actually the ethnicity of the people who are being or have enrolled in the trials in a way to hopefully mitigate some of these issues. But, again, we still have a long way to go. Some of the stuff we’ve covered a couple years in a row, because we want to see if there’s been advances in a year. Some of them, yes. A lot of them, unfortunately, there hasn’t been any. I think should we do this again? Yes, we probably will. Most of the people who’ve at least given us feedback, enjoy it, think it’s worthwhile. Let’s think ahead to Team PAH 2025. We’ll put out the topics, but it’s up to the people who are out there to send in abstracts to fill those topics. Then, we decide who we’re going to pick, what the order, stuff like that, which I think that’s clearly unique. There’s no other symposium that does it this way. Other symposiums, which I either do or organize or whatever, you pick a topic, you go, “Okay, we’ll invite Joe Blow,” and whatever. This is very different. We’re really dependent on the abstracts that people send in. We make that pretty clear. I think I said that by closing arguments. If you don’t like the topics, blame yourself because you send in the abstracts. I’m Hap Farber. I’m aware my patients are rare. Learn more about pulmonary hypertension trials at . Follow us on social @phaware Engage for a cure: #phaware Share your story: #phawareMD /episode/index/show/phawarepodcast/id/34683220

Episode 511 - Hap Farber, MD 03/10/2025

Drs. Kevin and Heather Shannon - phaware® interview 510 03/04/2025

Drs. Kevin and Heather Shannon - phaware® interview 510 Co-Founder and Board Member, Drs. Kevin and Heather Shannon share the inspiring story of — a transformative summer camp for children with heart disease. Hear how one patient’s journey ignited a movement to create a safe, empowering space where kids can embrace their scars, form lifelong friendships, and experience the joy of camp without limits. From the challenges of the first year to the life-changing moments that keep them going, the Shannons share how Camp del Corazon has touched countless lives and redefined what it means to live fully with a medical condition. The Shannon’s will be honored at on April 5th, 2025 at the Skirball Center. Get your tickets or participate in the online auction . #galadelsol #campdelcorazon #heartcamp #camplove Kevin Shannon, MD: My name is Kevin Shannon. I am a pediatric cardiologist and the co-founder and medical director of , a residential summer camp for children affected by heart disease. Heather Shannon, MD: I am Heather Shannon. I am also known as Dr. Heather Vandeweghe. I am a pediatrician, married to Kevin Shannon. I am now retired… happily. Kevin Shannon, MD: I finished my pediatric cardiology fellowship in 1993, and had an interest in treating abnormal heart rhythms, something called electrophysiology at the time. It was a very new subspecialty in pediatric cardiology. My boss happened to be the person who did that, Tom Klitzner, who became Director of Pediatric Cardiology in 1995. He was an electrophysiologist and had recruited me into his program. Around the time that I was finishing my fellowship, Tom had asked me to see a patient for him, and it was this patient who triggered the start of Camp del Corazon. It was a young man who had a fairly common valve abnormality, his version of it was fairly severe. He had deteriorated fairly rapidly from the age of about five to the age of about eight. At age five, he was pretty normal, played soccer, ran around, was in normal school. By age about eight, he had dropped out of school because he was so fatigued with any kind of exertion. He'd reached a point where he was being carried from his home to the car to go places. His doctor, at the time, was very old school and didn't believe in heart replacement therapy for children, because just a decade prior, the survival of that was very poor. He allowed this young man to get very limited, to the point that he developed arrhythmias. He got referred to my boss, who asked me to see him. I saw this young man and my first impression was, this is a bad valve and we're not going to make any progress in him until we fix the valve. But the parents clearly were resistant to that. So, we focused instead on the arrhythmia. We did a procedure called an ablation, where we eliminated his arrhythmia, where he happened to meet Lisa Knight, who was what we called the “arrhythmia nurse” at the time. She scheduled all of our procedures and then assisted with them. He did a little bit better with the arrhythmia. I saw him several times, and eventually convinced the parents he should have heart surgery and get this valve replaced. I had them meet with the surgeon and they finally agreed. He was in and out of the hospital in a week. He just flew through the surgery, because his heart was so much better having a functioning valve that the fact that he'd needed heart surgery and been on bypass was of no consequence to him. Then, every time I saw him after that, the parents were like, "Oh my gosh, he can do this now. Oh my gosh, he did this now." He had his surgery in January of 1994, and then in September of '94, he was back in school. He was playing soccer and was doing great. I'd literally been seeing him every few weeks, because I didn't have that many patients, and I was very worried about him. But then, come September, he was doing really well, so I didn't see him for a while. I saw him for his one-year follow-up in January. I went to listen to him and he wouldn't take his shirt off. Instead, he pulled his arms inside and just held the shirt out so I could reach up underneath and listen to him. I asked him, "Are you doing that because you're self-conscious about your scar?" Before he could answer, his mother said, "I haven't seen his scar since he was well enough to put his own shirt on." So, his own mother hadn't seen this scar in months. I asked, "Is that because kids at school tease you?" He said, "No, no one at school has ever seen it except my best friend, and he begged me to show it to him." I said, "So how did you change your clothes at school?" He proceeded to show me how he could change his clothes by putting one shirt on over the shirt he had and then pulling the previous shirt out through the neck hole, never exposing his chest. As I watched him go through this contortion to do this, I was kind of devastated, because I thought I'd done so much for this child and made him so normal. What I really did is I gave him a different problem that he saw every single day in the mirror and was just afraid to show anybody else, including his mother. That was a very discouraging event for me, because I was so proud of myself up until then, and then I realized, hey, I didn't make this child as normal as I thought. Around that time, I got an email from a listserv. Emails were brand new in 1993, and I had just signed up for this thing called PediHeart, where you would get email blasts from people who posted on it. I got this post from someone about a camp in Louisiana called Camp Bon Coeur. A lot of people responded to the post about how great the program was and how much it helped kids feel better about their scars. Most of the talk was about kids who get to see other kids with scars, they feel better about it. I wrote away for information. I set everything up for this kid to go to this camp. I had the local Mended Hearts to sponsor him, I had the application, I had everything all lined up, all I had to do was get the mother to fill it out and we would be on our way. They came to see me around April of that year, I had a big packet of stuff. I brought it to clinic and I was going to give it to them. I explained what it was and where it was, and the mother looked at me and she's like, "Louisiana's 1,500 miles from here!" There was no way she was going to send this child, who a year earlier, she'd been carrying from the car to the house. There was no way she'd let him go to Louisiana for a week. So, they didn't have any interest in it, didn't want any of the stuff. So, I had all this stuff and I brought it back to my office and put it on my desk. Lisa Knight saw it there when she came in to talk about scheduling patients, and I explained to her why I had it. She had a relationship with the people who own Catalina Island Camps, or the people who ran it. She looked at me and she said, "We can do that," and she picked up the phone. She called Tom and Maria Horner, who were the directors of Catalina Island Camps at the time, and told them we wanted to start a summer camp for children with heart disease, what did they think. They needed a community service component for their lease on Catalina. So, they were like, "Oh, yeah, we can do that. Let's do it over Labor Day," which was Labor Day of 1995. So, in April of '95, we were Camp Directors planning to go to the island in August. That was the start of what was a much more complicated process than I thought. But the short version is we were able to raise money asking vendors and people who sold products to the hospital, in particular, cardioplegia, because one of the surgeons got involved. We raised $15,000 and we arranged to take a boat to Catalina Island, use their site for three days, and then take it back. We recruited kids by going through the database at UCLA and finding children who were still alive and who definitely had heart disease, because I didn't want to invite anybody who'd lost a child and I didn't want to invite anybody who'd been told their child doesn't have a heart condition. The thing I remember most about that process was when we wrote the letter inviting people, we said, "There are two equally important goals of this program. The first is to allow children with heart conditions to experience something that other children take for granted, a residential summer camp experience. The second is to allow children with heart conditions to interact with peers, children who have the same experiences and concerns that they do." That was the birth of Camp del Corazon. Heather Shannon, MD: When he came home from work and mentioned this, I was just over six months pregnant and had a one and a half year old. I was like a deer in the headlights and said, "Sure, whatever." My father had started summer camps, mainly so he knew exactly where his children were going when I was younger, so I was not unfamiliar with being part of a family that started a camp and were Camp Directors, but really had no idea that we were stepping into such a lifelong process. The best part about it was that we were going to a campsite that was willing to support us in the sense that they kept all their counselors to do the activities, so we didn't have to find counselors, which was huge that first year. Basically, our job was just to raise enough money and then get the children from UCLA Medical Center on a bus to the dock, get them on the boat, get them through the boat ride to get to Catalina Island. Once there, the Directors of Catalina Island Camps pretty much took over and did a lot of the legwork. Those three months, four months, that we worked on it didn't seem that hard. Lisa Knight did most of the recruiting of campers and getting the information she needed, which, at the time, was a lot less than what we were require now. We obviously didn't realize the amount of work it takes to deliver all the medications for these kids through a three-day stay on the island, but we learned very, very quickly. Kevin Shannon, MD: There’s lots of serendipity with camp. The biggest one was obviously the first year, we had a site willing to give us staff, who knew how to do all the things that counselors have to do. To be honest, Tom and Maria Horner, every time we'd sit down and talk to them about philosophies of child raising and discipline and running camps and things, we are spot on on the exact same page with everything. Having them as partners in this was huge. The first year, we had no idea what we were getting into, to be honest. We knew we needed to make this safe. We had as much medical information as we could possibly gather about these kids. We actually took any medical person that was willing to come. We said, "Fine." We told them they could bring their kids, because at the end of the day, we only had 49 campers who got on the boat. We only had 84 who filled out applications. We knew we needed more than that number to have momentum in groups of kids. So, we let any staff that wanted to come with their kids that were in the age range. We put them in cabins with these other kids the first year. It was kind of a whirlwind from we'd get them all on the bus, we had to drive to Newport Harbor, because the only boat we could get was a charter out of Newport Harbor. We had to carry all the luggage three blocks from the bus. Then, that was '95, there was a hurricane off Hawaii, but it was sending 15-foot swells all the way to Santa Monica Beach, so we rode out in these huge swells and almost everybody got seasick. So, we got through those three days. Those are really, in all honesty, still a blur in my mind. My most silly memory is I remember wandering around between cabins late at night after everybody had gone to bed just listening to see if anybody was seizing, because I had in my brain, if anybody had a problem, the first thing they would do is seize and make noise. I don't know why. But nobody ever has had a problem at night, knock on wood. Heather Shannon, MD: After the first year, many friends of Lisa Knight came on as counselors. We realized that we had to start having our own counselors. The first year, which would've been the second year of camp, finding enough people was a little bit of a struggle. Catalina Islands Camps gave us some more staff, and we mustered through. Then, once word got out about their experiences, these counselors, we became inundated with people who wanted to be counselors at this camp. Counselors of other camps that had ended by Labor Day, they wanted to come. They wanted to be a part of it. It just flourished to the point where we started to have to have a real application process and a waiting list for counselors, and that has continued to this day. Fortunately, after about 10 years of camp, a lot of the campers grew to an age, because we require them to be over 20, where they could be counselors. That was a whole other process and place to get counselors. We've enjoyed that tremendously, to see these children grow up and become great people and do great things, which normally we envision them thinking that they can't do anything. Then, they come back and are counselors and such great support systems. So now, probably about 30% or 40% of our counselors are former campers of some sort. Kevin Shannon, MD: I've had many parents tell me, "The hardest thing I've ever done is watch my child get on that boat." Then they say, "But after they got back, I knew it was worth it." For parents, it's hard because this is a child that they've seen go through surgeries, that they've slept in the hospital at the bedside, that they've gotten permission slips and paperwork to the schools and not really let them out of their sight for very long. For about 70% of our campers, their first night at camp is their first night with anybody but a relative. So, it's a pretty big ask, and I tell our counselors all the time, the thing we market isn't a summer camp, it's trust. We ask these parents to give us their most prized possession, who's 1,000 times more fragile than anybody else's most prized possession, for four nights and five days, and to make sure that not only are they getting their medicine and they're being watched carefully, but that if something goes wrong, we're prepared. That's really what we want to let people know or feel. We don't market it in the sense we don't tell anybody that, but we try and give that impression and make sure people know that we do training. We find out as much as we need to know about their children. We have emergency action plans. What we want them to know really is their child's going to come and have a summer camp experience, it's not going to be about their heart disease. Heather Shannon, MD: As all children are, they're very conscientious about the scars and the marks and such, partly because I think it brings back memories of what they went through getting those scars. But we found at camp, there is no hiding scars. After about an hour or two, there are scar contests, there's by number how many scars you have, who has the worst scar, who has the biggest scar, et cetera, et cetera. Now, they enjoy that with the other counselors who have been campers prior. I think that they don't stare at each other's scars, because they are used to seeing those scars and such. It makes them feel very comfortable that they can be around other children and see that other people have these scars. I think many of these children think they're the only one in the world that has this scar and they haven't seen anybody else with it. That just puts them part of the group and children want to belong. I think the scar becomes a fairly large component of this whole process at camp. Kevin Shannon, MD: The first three days of camp were a whirlwind for me, I don't remember doing anything. I know I kayaked, I know I helped kids in and out of kayaks. I know that I had been a lifeguard. We had lifeguards, but I was on the waterfront a lot. I was wandering around a lot, just checking on things. We come back and I was exhausted. We got all the kids back to their parents. We, of course, had one child that the parents were four hours late. So, finally got him off with his parents, go home, get some sleep, and then Tuesday morning, I went back to work. I remember very distinctly, I got a call from a mom, she says, "Dr. Shannon, I just had to call and tell you that you wouldn't believe Carly when she came back! You brought back a different child. She was so excited about her heart disease and so excited about her scar and so excited about all the things she got to do. We can't wait for next year!" That was the first time it dawned on me that we were going to have to do this again. That kind of enthusiasm for “you brought back a different child,” really struck me. The next event like that I recall is I got an email from the mother of one of my patients. It was a very long, well-written email. It basically said, "Dear Dr. Shannon, I just want to let you know this summer camp was amazing. We sent you a child who was ashamed of his heart disease, who looked at the ground all of the time, who didn't see a future, didn't look forward to a future. You brought back a child who held his head up high, who is proud of what he's been through, who really saw a future and an exciting one. We can't thank you enough for doing that for our child. It's something we never could have done." That has been the raison d'etre of camp, is to have that kind of transformative change on children. There's lots of benefits to summer camps Even children without special conditions benefit from them. The biggest one probably being the friends that you make. For a lot of these kids, they feel a little bit outcast or so different from other children. They don't make friends easily, but they come to camp and they make friends that they then keep in touch with the rest of the year and they look forward to seeing every year when they come back. Heather Shannon, MD: I remember the first year, Kevin running to the ATM machine to get money out of our account to pay for the bus. I remember the buffalo walking through camp and everybody going, "Well, what do we do with a buffalo in camp? How do we get rid of it? It's awfully big." I'll have to say, most recently, the hurricane that came through, there was lots of parents very frightened about that, but we had a great time on the island. Rained a little bit, there were water streams and mud everywhere. We had an extra day on camp, so we had to come up with an activity for that rainy day and the counselors did the most amazing thing and I would say one of the best days at camp we had with the kids. The counselors are amazing. They make things happen. There's just been so many magical moments, things that we do every year, disco bingo. It's just something that is loved by everybody. We used to do something called a snipe hunt with the little kids, where they went out in the evening and we usually had Kevin and some other leadership were out there with two flashlights with red over the ends of it like the eyes of these little monsters. The kids would be screaming and such. Every year, there's always something special and fun to watch. Kevin Shannon, MD: From the very start, we had this recognition that if you have a child with heart disease, regardless of your financial situation, it's a huge burden. For many families, the vast majority, it's a huge financial burden. Even if you have insurance that covers all the medical costs, there's lost days at work, there's extra travel, there's nights in hotels, there's copays, there's all kinds of expenses and loss of income associated with it. The last thing we wanted to do is create any kind of financial burden for these parents, so from the get-go, it was free. The model for Camp Bon Coeur was you would get local Mended Hearts organizations to sponsor it, which for one kid, $1,500, our local Mended Hearts could do that pretty easily. $15,000 was a... /episode/index/show/phawarepodcast/id/35098600

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