Global Hemophilia Report

In 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

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Hemophilia 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. <...

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 treatment, the experts underscore the enduring importance of ongoing research in understanding and preventing FVIII inhibitors.

Contributors:

Glaivy Batsuli, MD

Bhavya Doshi, MD

David Lillicrap, MD

Carol Miao, PhD

Kathleen Pratt, PhD

Radek Kaczmarek, PhD

Senior Advisor: Donna DiMichele, MD

Hosted & Written by: Patrick James Lynch

Featured Advertiser:

Sanofi

Subscribe to the Global Hemophilia Report

Show Notes:

Listen to the Global Hemophilia Report Episode 1: Inhibitors: Prevention, Eradication, and Lived Experiences

Conflicts of Interest: Dr. Kathleen Pratt is an inventor on patents related to factor VIII immunogenicity, and she has received research funding in the past from pharmaceutical companies that manufacture human FVIII. She has no other relevant conflicts to report.

Disclaimer: The contents of Dr. Pratt's presentation are the sole responsibility of the speaker and do not necessarily reflect the views, opinions or policies of Uniformed Services University of the Health Sciences (USUHS), The Henry M. Jackson Foundation for the Advancement of Military Medicine, Inc., the Department of Defense (DoD) or the Departments of the Army, Navy, or Air Force. Mention of trade names, commercial products, or organizations does not imply endorsement by the U.S. Government.

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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 & Written by: Patrick James Lynch

Featured Advertiser:

Sanofi

Subscribe to the Global Hemophilia Report

Show Notes:

Presenting Sponsor: Sanofi

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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 Global Hemophilia Report

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:

BloodStreamMedia.com

BloodStream on Facebook

BloodStream on Twitter