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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Gene therapy, hemostatic rebalancing therapies, memetic therapies, and even factor replacement therapy are all currently being explored in clinical trials, but while these therapies are paving the way to historical treatment ease and efficacy, they are also sweeping the hemophilia community into uncharted territory of potentially new challenges that must be addressed. This episode explores the current state of novel therapy investigation in hemophilia.

Contributors:

Dr. Maria Elisa Mancuso

Dr. Glenn Pierce

Dr. Steven Pipe

Dr. Lindsey George

Dr. Margareth Ozelo

Dr. Guy Young

Dr. Michael Recht

Dr. Rich Gorman

Mark Skinner

Luke Pembroke

 

Senior Advisor:

Dr. Donna DiMichele

Links to learn more:

• Future Therapies - National Hemophilia Foundation
• 2021 clinical trials update: Innovations in hemophilia therapy
• Advances in the management of haemophilia: emerging treatments and their mechanisms
• Emerging drugs for hemophilia A: insights into phase II and III clinical trials
• Discussing investigational AAV gene therapy with hemophilia patients: A guide
• Hemophilia Clinical Trials - ClinicalTrials.gov

Show Notes:

Featured Advertiser: Sanofi

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Inhibitor development has long been considered the most significant complication of severe hemophilia, but with the licensure of the first non-factor therapy and a robust pipeline of novel and gene therapeutics being investigated, is it still of critical importance that we better understand inhibitors? If so, what research is currently taking place, and what can he hope to learn from it? This episode of the Global Hemophilia Report from BloodStream Media features contributions and citations from investigators, clinicians, and research funders dedicated to this topic, as well as from patients and caregivers who can speak to their lived experience of inhibitors.   For more on inhibitor research:

• Inhibitors and Hemophilia - CDC
• ATHN 8: PUPs Matter Study - ATHN
• The Hemophilia Inhibitor Eradication Trial - ClinicalTrials.gov
• A New Plan for Tackling Inhibitors - NHF
• ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders - ASH
• The prospective Hemophilia Inhibitor PUP Study reveals distinct antibody signatures prior to FVIII inhibitor development - ASH

  Show Notes:

Featured Advertiser: Sanofi Genzyme

Subscribe to BloodStream Media’s Global Hemophilia Report

Senior Adviser: Dr. Donna DiMichele

 

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 

Led by science, curiosity, and storytelling, the Global Hemophilia Report from BloodStream Media is an entertaining monthly journey through the investigations and science driving hemophilia research around the world.

Produced by: Believe Limited & BloodStream Media Hosted by: Patrick James Lynch Senior Advisor: Dr. Donna DiMichele Featured Advertiser: Sanofi Genzyme

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