A Global Look at Eosinophilic Disorders: Unmet Needs

Co-hosts Ryan Piansky, a graduate student and patient advocate living with eosinophilic esophagitis (EoE) and eosinophilic asthma, and Holly Knotowicz, a speech-language pathologist and feeding specialist living with EoE who serves on APFED’s Health Sciences Advisory Council talk with guest Florence Roufosse, MD, PhD, Professor of Medicine, Internist and Clinical Immunologist at CUB-Hôpital Erasme, Brussels, and President, International Eosinophil Society.

In this episode, Ryan and Holly discuss with Dr. Roufosse some of the many unmet needs related to eosinophil-associated diseases. The discussion covers diagnosis, treatment, access to multi disciplinary care, research, and awareness. Dr. Roufosse identifies specific questions she sees an urgency to address and discusses clinical trials. Listen in for a better understanding of some of the unmet needs of patients with eosinophil-associated diseases.

Disclaimer: The information provided in this podcast is designed to support, not replace the relationship that exists between listeners and their healthcare providers. Opinions, information, and recommendations shared in this podcast are not a substitute for medical advice. Decisions related to medical care should be made with your healthcare provider. Opinions and views of guests and co-hosts are their own.

 

Key Takeaways:

[0:50] Ryan welcomes co-host Holly Knotowicz. Holly introduces the topic: a global look at eosinophilic diseases and unmet needs.

 

[1:28] Holly introduces Dr. Florence Roufosse. Dr. Roufosse is a professor of medicine, an internist, and a clinical immunologist at Erasme Hospital in Brussels and is President of the International Eosinophil Society.

 

[1:43] In addition to her clinical work, Dr. Roufosse leads translational research projects to improve the understanding and treatment of hypereosinophilic syndromes (HES). She is also involved in international clinical trials evaluating treatment options for patients living with HES and eosinophilic granulomatosis with polyangiitis (EGPA).

 

[2:18] Dr. Roufosse is an internist working in an academic hospital. An internist is a diagnostician, whom people see if they have many complaints that involve various organs to find a unifying diagnosis for their problems.

 

[2:47] Dr. Roufosse’s interest in eosinophils started when she met Dr. Elie Cogan. He had just published a case report on a patient with hypereosinophilic syndrome who also had T-lymphocytes present. The T-cells were producing growth factors for eosinophils.

 

[3:21] The hypereosinophilic syndrome in the patient was due to these abnormal T-lymphocytes. The case was in 1996. Dr. Cogan asked Dr. Roufosse if she would agree to do a doctoral fellowship on the topic and try to dive deeper into understanding the disease in this patient.

 

[3:37] Dr. Roufosse has been working on HES ever since, doing translational research, working with blood samples from patients she sees in the clinic, and studying the cells functionally and on a molecular basis. Dr. Roufosse is foremost a clinician who sees patients in the clinic every day.

 

[4:00] Dr. Roufosse regularly gets referrals for HES and EGPA. She has a large cohort of patients. Her main job consists of reaching a diagnosis and giving the best treatment to these patients.

 

[4:44] The main mission of the International Eosinophil Society (IES) is to bring together clinicians, investigators, and researchers interested in eosinophils and eosinophilic disorders. The society held its first meeting in 2001. The pandemic prevented them from celebrating their 20th anniversary in person, but they will meet this summer.

 

[5:20] IES brings together clinicians who see patients and people who investigate eosinophils in the lab. Close interactions between clinicians and investigators drive the motivation to understand the biology of these disorders, find the therapeutic targets, and work together, combining findings to make progress.

 

[6:52] There are common unmet needs for eosinophilic diseases and specific unmet needs for individual diseases. Transitioning from pediatric to adult care is an unmet need in eosinophilic esophagitis because the disease begins in childhood. However, eosinophilic syndrome and EGPA occur most commonly in adults.

 

[7:51] Diagnosis is a bottleneck. When people get the right diagnosis, they are already well on their journey of receiving treatment. Diagnosis combines the need for increased awareness among patients, the public, and physicians. A delayed diagnosis may have consequences for patients, such as a progressive disease that is not controlled.

 

[8:31] High levels of eosinophils have the potential to damage organs and induce functional damage and functional alterations. They favor fibrosis. The longer eosinophils stay at high levels, the more harm can occur, so there is a consequence in delayed diagnosis.

 

[8:55] Patients with high eosinophils in blood and tissues will often receive corticosteroid therapy. This is good for a few days but there are numerous side effects to corticosteroid therapy in the short-, middle-, and long-term. Also, the longer the journey is to diagnosis, the more people get discouraged and depressed.

 

[10:02] Part of the unmet need in diagnosis is awareness. There’s a saying, “What you don’t know, you can’t diagnose.” Many physicians don’t know about these eosinophilic conditions.

 

[12:13] APFED and Dr. Roufosse worked together to author “Improving Care in Eosinophil Associated Diseases: A Charter.” The charter outlines the needs and the rights of patients with eosinophil-associated diseases.

 

[13:03] The rights of patients include the right to a timely diagnosis, which implies awareness about the diseases among the public, healthcare providers, and policy makers. Patients also have the right to access multi disciplinary care teams, as the disease may affect different tissues and organs. A patient may even have several disorders at once.

 

[14:06] It’s important that if treatment options are available; patients need to have access to them. This is not the case in all countries. Patients need rapid access to the therapies once an indication has been decided by a specialist.

 

[14:53] Quick diagnosis will decrease the accrual of damage due to the disease, inadequate treatment, and overexposure to corticosteroid treatment. From a psychological point of view, it’s very reassuring to rapidly feel that a label has been given to the disease and that there’s a plan. When there’s no label, there’s no plan.

 

[15:47] The key to shortening the diagnostic timeline is awareness at all levels of healthcare education and specialization. General practitioners need to be educated on what it means and what to do when they see a high eosinophil count in blood or tissue. There needs to be access to the tools to diagnose the associated disorders.

 

[17:00] Patients need easier access to referral centers where they see the experts and access the tools for diagnosis. Multidisciplinary team care is needed when diseases hit different organs.

 

[17:44] When EGPA is controlled with immunosuppressive therapy, often asthma remains a serious problem and very difficult to manage. You need a pulmonologist to take care of that aspect. You need an ear, nose, and throat doctor for complications in those areas. Cardiovascular complications need a cardiologist.

 

[19:21] Dr. Roufosse says it is still challenging for patients to access a multidisciplinary care team. Creating a multidisciplinary care team requires resources. It has to be supported by a hospital. The institution needs to be an academic institution with funding, as doctors meeting to discuss a difficult case does not generate income for the institution.

 

[20:39] Ryan worked with a multi disciplinary care team as a young EoE patient. He traveled every year from his home in Georgia to the multi-disciplinary care team in Denver, Colorado. The fact that his family was able to travel out of state was wonderful. The team was effective and he misses it. Now he has separate appointments for each type of doctor.

 

[22:32] New therapies, for those who have had access to them through clinical trials, are decreasing the need for corticosteroid therapy in many patients. Practically half of the patients with hypereosinophilic syndrome can be tapered off corticosteroids. Dr. Roufosse tells of one of her first patients, whose life was changed by these therapies.

 

[24:04] Not every aspect of disease responds as well to these new therapies, revealing new unmet needs that are being tested with additional new therapies. Dr. Roufosse’s first patient has been helped greatly with biologics for almost 20 years now.

 

[24:55] Dr. Roufosse speaks of more unmet treatment needs. Eosinophils are only a part of the picture. EoE is a complex disease about more than eosinophils. We need more understanding of what the key pathogenic events are across the range of eosinophil-related disorders to be able to identify new therapeutic targets.

 

[26:00] With clinical trials, it can be difficult to find suitable endpoints for the trial and define the efficacy of a new treatment. When is the disease still active with treatment? When can we start decreasing the intensity of treatment? How long do we have to treat with these drugs? Many of these questions are still completely unanswered.

 

[26:53] Over the past 25 years, the time to diagnosis has decreased. Dr. Roufosse rarely now has a patient referred to her that already has severe, irreversible damage.

 

[27:21] How to score disease activity is an area where we still have a lot of work to do.

 

[27:37] Medical codes are used to classify and group diseases for reporting statistical information. In the U.S., they are important for diagnostics and what treatments you get access to. In 2020, new codes (ICD-10) were approved for various subsets of eosinophilic diseases.

 

[28:09] ICD-10 codes are used less in Belgium than in the U.S. but a disease does not “exist” if it doesn’t have a code. Without a code, there is no access to diagnostic tools or reimbursement for therapy for the disease. You need codes to justify hospitalization. C codes also allow Holly’s patients to receive feeding therapy.

 

[30:43] To help patients and caregivers identify medical professionals offering care for eosinophil-associated diseases, APFED hosts a Specialist Finder on its website. It largely has clinicians in the U.S. You can access it at apfed.org/specialists. There is also a badge for specialists who are members of the International Eosinophil Society.

 

[31:10] Dr. Roufosse suggests having handouts for patients listing patient associations and places to find information on their disease. Some people are more comfortable with something tangible and physical. Not everyone uses Google! Doctors need to use different means to share information on how to access specialists.

 

[32:11] Policymakers are important in bringing together information to give access to proper care for these disorders.

 

[33:56] Dr. Roufosse discusses unmet needs in research. We understand so little about these diseases, although great progress has been made in understanding that the eosinophil is a toxic cell that can cause damage and if you target those eosinophils, you are going to prevent some of that damage. We don’t yet have the full picture.

 

[34:32] Some unmet needs are understanding what part of each of these disorders is influenced genetically or combines genetics with environmental triggers. What cells initiate the inflammatory process? What cells and mediators maintain the process? Which mediators should we target? How do we do it without compromising immunity?

 

[35:12] It appears that targeting eosinophils in humans is innocuous, but what are they for? We are learning about what eosinophils are for in mice, but very little about what they’re for in humans. Are normal eosinophils irreplaceable or are there redundant functions with other cells? Some patients have high eosinophils without any illness.

 

[35:46] Some patients have lower eosinophil counts than others and they have rapidly progressive cardiac damage. What characterizes a bad eosinophil? We don’t have the tools to recognize that, yet. We can’t predict which patients are going to require more aggressive therapy earlier in their disease course. That’s very important to research.

 

[36:39] Dr. Roufosse recommends using the apfed.org website with its Specialist Finder and relevant patient resources. Awareness is the key to faster diagnosis, accessing treatment, and avoiding harmful treatment.

 

[37:35] Ryan and Holly thank Dr. Roufosse for being on the podcast. Ryan shares the links including apfed.org, apfed.org/specialists, and apfed.org/eos-connections links. Holly and Ryan thank APFED’s education partners, linked below.

 

Publications discussed:

• Patient charter: Apfed.org/advocacy/ead-patient-charter/ 
• Link.springer.com/article/10.1007/s12325-022-02110-8
• Pubmed.ncbi.nlm.nih.gov/29672914/

 

Mentioned in This Episode:

American Partnership for Eosinophilic Disorders (APFED)

APFED on YouTube, Twitter, Facebook, Pinterest, Instagram

CUB-Hôpital Erasme, Brussels

Episode 18: “Transition of Care from Pediatric to Adult Providers, with Dr. Giresh Hiremath”

Episode 15: “Access to Specialty Care for Eosinophilic Esophagitis (EoE) with Dr. Emily McGowan”

“Improving Care in Eosinophil-Associated Diseases: A Charter”

apfed.org/specialists

Real Talk: Eosinophilic Diseases Podcast

 

Education Partners: This episode of APFED’s podcast is brought to you thanks to the support of Bristol Myers Squibb, GlaxoSmithKline, Mead Johnson Nutrition, Sanofi, and Regeneron.

 

Tweetables:

 

“The International Eosinophil Society is a dynamic ... society whose main mission is to bring together clinicians, investigators, and researchers ... interested in eosinophils and eosinophilic disorders. ” — Florence Roufosse, MD, PhD

 

“Transitioning from pediatric to adult care is typically an unmet need in eosinophilic esophagitis, which very commonly begins in childhood.” — Florence Roufosse, MD, PhD

  

“The unmet need, in … diagnosis, is awareness. … There’s a saying that what you don’t know, you can’t diagnose. … Many physicians don’t know about these eosinophilic conditions. … Turn toward someone … who is more likely than you to know.” — Florence Roufosse, MD, PhD

 

 

Featured speaker:

Florence Roufosse, MD, PhD

Professor of Medicine, Internist, and Clinical Immunologist at CUB-Hôpital Erasme, Brussels, President, International Eosinophil Society

 

Dr. Florence Roufosse is a Professor of Medicine, Internist, and Clinical Immunologist at CUB-Hôpital Erasme, Brussels, and is President of the International Eosinophil Society. She is in charge of a specialized consultation dedicated to diagnosing and treating eosinophil-related conditions, that is integrated in the European Reference Network: EuroBloodNet. She also manages patients with systemic auto-immune and auto-inflammatory conditions.

 

Besides these clinical activities, Dr. Roufosse leads translational research projects to improve the understanding and treatment of lymphocytic variant hypereosinophilic syndrome (HES) and participates in international research efforts to better delineate disease course and treatment responses of HES. She is involved in the design and conduct of international clinical trials evaluating the efficacy of novel treatment options in patients with HES and eosinophilic granulomatosis with polyangiitis (EGPA), as well as sub-studies that aim to identify biomarkers and/or disease variants predicting treatment responses.