VJHemOnc Podcast cover image

VJHemOnc Podcast

Hemophilia updates from ISTH 2024: ongoing clinical trials, emerging gene therapies & remaining challenges

Aug 19, 2024
Omolade Awodu from the University of Benin discusses the latest clinical trials in hemophilia, spotlighting innovative treatments like Consissimab and Fiti Suran. Radek Kaczmarek from Indiana University delves into the advancements in gene therapies, including the use of adeno-associated viral vectors and challenges in delivering transgenes. Matteo Luciani shares insights on the hurdles patients face, emphasizing the need for long-lasting solutions to improve treatment adherence, especially for young children.
18:42

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Quick takeaways

  • Recent clinical trials have revealed promising investigational drugs, such as Consissimab and Fitucero, significantly improving bleeding control in hemophilia patients.
  • Emerging gene therapies show potential in stabilizing factor levels for hemophilia, yet challenges like variability in responses and accessibility remain critical issues.

Deep dives

Clinical Trials Show Promise for Hemophilia Treatments

Recent clinical trials have highlighted significant advancements in hemophilia treatments, particularly with the investigational drugs Consissimab and Fitucero. Consissimab, a two-factor pathway inhibitor, has shown efficacy in managing hemophilia in patients with both type A and B, demonstrating notably reduced bleeding rates compared to standard therapies. Similarly, Fitucero, which interferes with the production of antidermolein in the liver, has made strides in controlling bleeding and reducing rates in hemophilia patients. The promising results from these trials indicate a shift towards more effective management options and a potential move away from traditional factor replacement therapies.

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