Beyond the Bleed: Mastering Novel Therapies in Hemophilia
Treating the bleed is only half the battle. Detect arthropathy before it's permanent. Learn to apply novel prophylactic agents to prevent joint damage. Complete the activity and claim your FREE CME/NCPD/CPE/AAPA today: https://bit.ly/478dwcH View and download the slide deck here: https://www.slideshare.net/slideshow/... In this enduring webinar, originally presented @NBDFvideo annual meeting, Guy Young, MD @childrensLA, University of Southern California Keck School of Medicine) and Robert F. Sidonio, Jr., MD, MSc (Hemophilia of Georgia Center for Bleeding and Clotting Disorders, Children’s Healthcare of Atlanta), explore approaches including the importance of the interdisciplinary care team to remain up to date on advances and strategies to balance the benefits of therapy with patients’ quality of life, including improving early detection of hemophilic arthropathy. 00:00 – Introduction 02:44 – Patient case Hemophilia A 04:28 - Patient case hemophilia B 05:54 – Scope of the problem and unmet needs 14:34 - Musculoskeletal ultrasound 17:01 – Why do we need nonfactor therapies? 20:48 - Novel therapies 23:42 – Novel factor therapy: Efanesoctocog alfa 29:17 -Overview of factor VIII mimetics for hemophilia A 32:16 - Emicizumab 25:15 - Mim8 39:50 - NXT007 41:23 - Tissue Factor Pathway Inhibitors 42:38 - Marstacimab 54:12 – Fitusiran 1:11:06 – Patient case hemophilia A 1:11:55 - Patient case hemophilia B 1:12:09 – Valoctocogene roxaparvovec 1:13:06 - Etranacogene dezaparvovec 1:13:47 - Factors for therapy selection 1:15:49 – Polling Questions 1:19:16 – Key Takeaways Follow us here: Facebook / i3health Instagram / i3health Linkedin / i3health Soundcloud / i3-health X / i3health STATEMENT OF NEED Hemophilia is a rare bleeding disorder caused by a deficiency of coagulation factor VIII (hemophilia A) or coagulation factor IX (hemophilia B), resulting in spontaneous or post-traumatic bleeding episodes that can cause severe complications or even death. The treatment of hemophilia is traditionally based on preventing bleeding through expensive, lifelong infusion therapy with plasma clotting factors, recombinant factors, or monoclonal antibodies (Chernyi et al, 2024). Lack of effective prophylaxis can lead to subsequent disability and irreversible joint damage, known as hemophilic arthropathy. In recent years, the emergence of new therapies, including gene therapy, has revolutionized hemophilia treatment (Gualtierotti et al, 2025). DISCLOSURE OF RELEVANT FINANCIAL INFORMATION WITH INELIGIBLE COMPANIES i3 Health endorses the standards of the ACCME, ANCC, and ACPE that require everyone in a position to control the content of a CME/NCPD/CPE activity to disclose all financial relationships with ineligible companies that are related to the content of the CME/NCPD/CPE activity. CME/NCPD/CPE activities must be balanced, independent of commercial bias, and promote improvements or quality in healthcare. All recommendations involving clinical medicine must be based on evidence accepted within the medical profession. Relevant financial relationships exist between the following individuals and ineligible companies: Guy Young, MD, discloses that he has served as a consultant for Alnylam, ASC Therapeutics, Bayer, BioMarin, CSL Behring, Genentech/Roche, HEMA Biologics, Novo Nordisk, Octapharma, Pfizer, Sanofi, Spark, and Takeda; and that he has received grants/research support from HEMA Biologics and Sanofi. Robert F. Sidonio, Jr., MD, MSc, discloses that he has served on an advisory board or panel for Bayer, HEMA Biologics/LFB, Hemab, Novo Nordisk, Octapharma, Pfizer, Sanofi/Sobi, Star, and Takeda; that he has served as a consultant for Bayer, HEMA Biologics/LFB, Hemab, Novo Nordisk, Octapharma, Pfizer, Sanofi/Sobi, Star, and Takeda; and that he has received grants/research support from HEMA Biologics/LFB, Octapharma, and Takeda. The i3 Health planners, reviewers, and managers have nothing to disclose. i3 Health has mitigated all relevant financial relationships. COMMERCIAL SUPPORT This educational activity is supported by an independent medical education grant from Sanofi. #medicaleducation #robertsidonio #guyyoung #hemophilia #bleedingdisorders #hematology #hematologist #bleedingandclotting #clotting #childrenshospitalUCLA #prophylaxistreatment #prophylaxis #meded #patientadvocacy #healthcare #medicine #science #learnonyoutube #doctor #premed #trending #educational #didyouknow #mindblown #inspiration #knowledge
