Prophylaxis: Protection, Achievements, and Shortcomings

Prophylaxis in hemophilia has led to extraordinary progress, however, recent studies suggest that although the overall musculoskeletal health of persons with hemophilia has significantly improved, the goal of attaining a level of musculoskeletal health comparable to that of the general population without hemophilia has still not been achieved. Furthermore, the impact of prophylaxis has been uneven across hemophilia populations and the promise of greater physical activity free of hemorrhage has not been entirely fulfilled. This episode explores the history, evolution, and current state of prophylaxis, and highlights the current and future areas of further investigation. Contributors:

Episode Advisors:

Dr. Marilyn Manco Johnson

Dr. Kathelijn Fischer

Contributors:

Dr. Robert Sidonio

Dr. Manuel Carcao

Dr. Maria Elisa Mancuso

Susan Lynch

Senior Advisor:

Dr. Donna DiMichele

Links to learn more:

• A history of prophylaxis in haemophilia
• Hemophilia A and B: Routine management including prophylaxis
• Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the Joint Outcome Continuation Study
• Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort
• Factor VIII replacement prophylaxis in patients with hemophilia A transitioning to adults: a systematic literature review
• Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies
• Optimal treatment strategies for hemophilia: achievements and limitations of current prophylactic regimens
• Optimising prophylaxis in haemophilia A: The ups and downs of treatment

Show Notes:

Presenting Sponsor: Sanofi

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