Hemophilia B & the story of Tsarevich Alexei - Part 1
Release Date: 10/13/2022
The Future of the Comprehensive Care Model
Global Hemophilia Report
The comprehensive care model for hemophiliathe is experiencing transformative evolution. Our panel of esteemed experts highlight how data and innovation are driving change across the globe, and share their insights on the challenges and advancements in advancing specialized care for individuals with hemophilia. Contributors: Amy Dunn, M.D. Sanjay Ahuja, M.D. Cedric Hermans, M.D., Ph.D., FRCP Jan Blatný, M.D., Ph.D Suely Rezende, M.D., Ph.D Senior Advisor: Donna DiMichele, MD Hosted & Written by: Patrick James Lynch Featured Advertiser: ...
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Chapters & Grassroots Advocacy
Global Hemophilia Report
This episode of the Global Hemophilia Report podcast explores the significance of grassroots advocacy within hemophilia associations and chapters, emphasizing the critical role of data in advancing advocacy efforts. Experts from various U.S. organizations discuss the historical impact of the 1980s blood contamination crisis and the evolution of advocacy through its challenges and triumphs. The conversation highlights the importance of data collection, transparency, and community engagement in sustaining and enhancing treatment and support for those affected by hemophilia. The episode also...
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The Evolving Landscape of Novel Therapies for Hemophilia: What’s Here & What’s Coming?
Global Hemophilia Report
On this episode we talk with experts Dr. Amy Shapiro, Dr. Maria Elisa Mancuso, Dr. Steve Pipe, Dr. Johnny Mahlangu, and Dr. Lynn Malec to delve into the ongoing evolution of hemophilia therapies. The discussion highlights recent advancements in treatments such as emicizumab, extended half-life factor VIII therapies, and investigates medications in clinical trials like Concizumab, Marstacimab, and Fitusiran. The episode also reflects on the role of treatment individualization and the need for more inclusive research data. Contributors: Johnny Mahlangu, MBBCh, MMed, FCPath Lynn Malec, MD,...
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Understanding & Preventing FVIII Inhibitors : An Enduring HA Research Priority
Global Hemophilia Report
On this episode of the Global Hemophilia Report, Senior Advisor Dr. Donna DiMichele leads a comprehensive discussion on FVIII inhibitors—a critical complication in Hemophilia A. The episode features experts such as Dr. Kathleen Pratt, Dr. David Lillicrap, Dr. Bhavya Doshi, Dr. Carol Miao, and Dr. Radek Kazmarek who explore the intricacies of immune responses, gene therapy, and novel therapies like emicizumab. They delve into factors like antigen-presenting cells, B cells, T cells, microbiome effects, and glycans that influence inhibitor development and tolerance. Despite advancements in...
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Lived Experience Experts: Taking Their Rightful Place at the Research Table
Global Hemophilia Report
In this episode of the Global Hemophilia Report, we delve into the crucial role of Lived Experience Experts (LEEs) in hemophilia research. Featuring a distinguished panel, the discussion covers the evolution, importance, and future of LEE involvement in research. The episode emphasizes the pivotal contributions of LEEs in making research more relevant and impactful, driven by personal and community experiences. Contributors: Len Valentino MD Samantha Carlson MSW, LMSW Randy Curtis Ray Stanhope Kyle Davis MD Senior Advisor: Donna DiMichele, MD Special Episode Advisor: Maria...
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Caregivers and Novel Therapies - Alleviating or Replacing Concerns?
Global Hemophilia Report
Our panel of subject matter and lived experience experts engage in a data-driven discussion about caregiver burden in hemophilia, historically, and how the rapidly evolving treatment landscapes impacts that burden. Contributors: Michelle Witkop, DNP, FNP-BC Kate Khair, PhD Beatriz Caceres, MD Kasha Lumsden, BSN, RN, RAC-CT Yasmin Pavri Senior Advisor: Donna DiMichele, MD Special Episode Advisor: Michelle Witkop, DNP, FNP-BC Hosted & Written by: Patrick James Lynch Featured Advertiser: Sanofi Subscribe to the Show Notes: Presenting Sponsor:...
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World Hemophilia Day
Global Hemophilia Report
On this episode of the Global Hemophilia Report, host Patrick James Lynch is on location in Madrid, Spain for the WFH 2024 World Congress. Patrick talks about the history of World Hemophilia Day and talks with some of the attendees at the congress. Contributors: Hazri Aris Johnny Mahlangu Rebecca, Hematology Resident Senior Advisor: Donna DiMichele, MD Hosted & Written by: Patrick James Lynch Featured Advertiser: Sanofi Subscribe to the Show Notes: Presenting Sponsor: Sanofi Subscribe to the Connect with the Global Hemophilia Report Connect...
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Translating Patient-Reported Clinical Outcomes into Patient-Informed Clinical Research Outcomes
Global Hemophilia Report
In this episode, delve into the transformative journey of patient-reported outcomes measures (PROMs) within hemophilia care, exploring their evolution from broad assessments of health-related quality of life to nuanced evaluations of physical ability, functionality, and psychological impact. Learn about pioneering instruments like the Hemo-TEM and the PROBE questionnaire, designed to capture the multifaceted burdens of hemophilia treatment and the patient experience. However, as the landscape of PROMs continues to evolve, discover lingering debates around their sufficiency compared to...
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Understanding Factor VIII & IX - In Coagulation and Beyond - How Much Are We Still Missing?
Global Hemophilia Report
The topic of discussion for this episode leans more toward the scientific side but has huge clinical relevance. For today, we will be asking our expert panel to lend their experience and expertise to the topic of FVIII and FIX, which we will be discussing from several related perspectives. Contributors: Radek Kaczmarek, PhD, MSc; University of Indiana, USA Peter Lenting, PhD; INSERM, Paris, France Ben Samuelson -Jones, M.D., PhD; CHOP Research Institute, USA Courtney Thornburg, M.D., MS; Rady Children’s Hospital, UCSD, USA Senior Advisor: Donna DiMichele, MD Hosted...
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Hemophilia Gene Therapy Redux – The End of the Beginning?
Global Hemophilia Report
Gene therapy for hemophilia A and B now exists as a licensed, prescribable treatment option for patients in certain countries; however, many questions and challenges remain. Senior advisor Dr. Donna DiMichele and patient-host Patrick James Lynch speak to global KOLs about the current state of hemophilia A and B gene therapies, both commercially and investigationally. Contributors: Lindsey A. George, MD Margareth Ozelo, MD Steven Pipe, MD Senior Advisor: Donna DiMichele, MD Hosted & Written by: Patrick James Lynch Featured Advertiser: Sanofi Subscribe to...
info_outlineHemophilia B is a rare inherited X-linked disorder characterized by a congenital anomaly in the factor IX gene leading to a variable deficiency in clotting factor IX. In this episode, we explore the known differences between Hemophilia B and Hemophilia A and discuss priority areas for further research into Hemophilia B. The episode also guides listeners on an entertaining journey through 19th and 20th century European monarchy and the unlikely role that hemophilia played in forever shaping Europe’s governance!
Contributors:
Bethany Samuelson Bannow, MD
Brian O'Mahony
Kathaleen M. Schnur, MSW, LCSW
Senior Advisor:
Donna DiMichele, MD
Episode Advisors (also contributors):
Dr. Amy Shapiro
Professor Jan Astermark
Hosted by:
Laurence Woollard
Links to learn more:
- Brown A. "The Royal Disease and The Royal Collapse: Political Effects of Hemophilia in the Royal Houses of Europe." Honor Scholar Theses. 2017;63. [Online]. Available at: https://scholarship.depauw.edu/cgi/viewcontent.cgi?article=1063&context=studentresearch [Accessed 10 October 2022]
- Hoffman TA. "Bad Blood: Hemophilia and Its Detriment to the Russian Imperial Family." Young Historians Conference. 2022;8. [Online]. Available at: https://pdxscholar.library.pdx.edu/cgi/viewcontent.cgi?article=1244&context=younghistorians [Accessed 10 October 2022]
- Lannoy N, Hermans C. The ‘royal disease’ – haemophilia A or B? A haematological mystery is finally solved. Haemophilia 2010;16:843-47. Doi: 10.1111/j.1365-2516.2010.02327.x
- Potts WTW. Royal haemophilia. J Biol Educ 1996;30(3):207-17. DOI: 10.1080/00219266.1996.9655504
- Price KD. "Diary of Nicholas II, 1917-1918, an annotated translation." Graduate Student Theses, Dissertations, & Professional Papers. 1966;2065. [Online]. Available at: https://scholarworks.umt.edu/cgi/viewcontent.cgi?article=3084&context=etd [Accessed 10 October 2022]
- Radcliffe J. “Rasputin and the Fragmentation of Imperial Russia.” Young Historians Conference. 2017;14. [Online]. Available at: https://pdxscholar.library.pdx.edu/younghistorians/2017/oralpres/14/ [Accessed 10 October 2022]
- Funnell APW, Crossley M. Hemophilia B Leyden and once mysterious cis-regulatory mutations. Trends Genet 2014;30(1):18-23. Doi: 10.1016/j.tig.2013.09.007
- Simioni P, et al. X-linked thrombophilia with a mutant factor IX (factor IX Padua). N Engl J Med 2009;361(17):1671-5. Doi: 10.1056/NEJMoa0904377
- Nogami K, et al. Clinical conditions and risk factors for inhibitor-development in patients with haemophilia: A decade-long prospective cohort study in Japan, J-HIS2 (Japan Hemophilia Inhibitor Study 2). Haemophilia 2022;28(5):745-59. Doi: 10.1111/hae.14602
- Thorland EC, et al. Anaphylactic response to FIX replacement therapy in haemophilia B patients: complete gene deletions confer the highest risk. Haemophilia 1999;5(2):101-5.
- Chitlur M, et al. Inhibitors in factor IX deficiency a report of the ISTH-SSC international FIX inhibitor registry (1997-2006). Haemophilia 2009;15(5):1027-31. Doi: 10.1111/j.1365-2516.2009.02039.x
- DiMichele D. The North American Immune Tolerance Registry: contributions to the thirty-year experience with immune tolerance therapy. Haemophilia 2009;15(1):320-8. Doi: 10.1111/j.1365-2516.2008.01880.x
- Astermark J, et al. The B-Natural Study – The outcome of immune tolerance induction therapy in patients with severe haemophilia B. Haemophilia 2021;27(5):802-13. Doi: 10.1111/hae.14357
- Iorio A, et al. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med 2009;171(8):540-46. Doi: 10.7326/M19-1208
- Soucie JM, et al. Occurance rates of haemophilia among males in the United States based on surveillance conducted in specialized haemophilia treatment centres. Haemophilia 2020;26(3):487-93. Doi: 10.1111/hae.13998
- Berntorp E, et al. Quality of life in a large multinational haemophilia B cohort (The B-Natural Study – Unmet needs remain. Haemophilia 2022;28(3):453-61. Doi: 10.1111/hae.14525
- Kihlberg K, et al. Treatment outcomes in persons with severe haemophilia B in the Nordic region: The B-NORD study. Haemophilia 2021;27(3):366-74. Doi: 10.1111/hae.14299
- Feng D, et al. Evidence of clinically significant extravascular stores of factor IX. Thromb Haemost 2013;11(12):2176-2178. Doi: 10.1111/jth.12421
- DiMichele DM, et al. Severe and moderate haemophilia A and B in US females. Haemophilia 2014;20(2):e136-43. Doi: 10.1111/hae.12364
- Buckner TW, et al. Management of US men, women, and children with hemophilia and methods and demographics of the Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study. Eur J Haematol 2017;98:5-17. Doi: 10.1111/ejh.12854
Show Notes:
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