Hemophilia B & the story of Tsarevich Alexei - Part 2
Release Date: 10/27/2022
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: ...
info_outline
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...
info_outline
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,...
info_outline
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...
info_outline
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...
info_outline
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:...
info_outline
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...
info_outline
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...
info_outline
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...
info_outline
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_outlineIn Part 2 of our Hemophilia B episode, we spotlight research priorities into women with hemophilia B, novel therapies to treat people with hemophilia B, and we conclude the story of Alexei Nikolaevich, the last Tsesarevich of Russia.
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]
- Gualtierotti R, et al. Updates on novel non-replacement drugs for hemophilia. Pharmaceuticals 2022;15(10):1183. Doi: 10.3390/ph15101183
- Hart DP, et al. International consensus recommendations on the management of people living with haemophilia B. Ther Adv Hematol 2022;13:1-22. Doi: 20406207221085202
- 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
- Nathwani A, et al. Liver gene therapy. Hum Gene Thur 2022;33:17-18. Doi: 10.1089/hum.2022.169
- O’Donovan M, et al. Recombinant factor IX-Fc fusion protein in severe hemophilia B: Patient-reported outcomes and health-related quality of life. Res Pract Thromb Haemost 2021;5(7):e12602. Doi: 10.1002/rth2.12602
- Peeling S. Russo-Japanese War. International Encyclopaedia of the First World War. 1914-1918. [Online]. Available at: https://encyclopedia.1914-1918-online.net/article/russo-japanese_war [Accessed 10 October 2022]
- 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]
Show Notes:
Presenting Sponsor: Sanofi
Subscribe to the Global Hemophilia Report
Connect with the Global Hemophilia Report
Global Hemophilia Report on LinkedIn
Global Hemophilia Report on Twitter
Global Hemophilia Report on Facebook
Connect with BloodStream Media: