Two Onc Docs
Hemophilia 2024 UPDATE
Aug 19, 2024
This week, explore the intricacies of hemophilia A and B, including their causes linked to clotting factors. Discover groundbreaking advancements in treatment, with a focus on recent gene therapies that are changing patient management strategies. Learn about the challenges of inhibitors and their implications in surgical contexts. Delve into diagnostic procedures and gain insight into tailored treatment options for acquired hemophilia, enhancing your understanding of this complex condition.
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Quick takeaways
- Hemophilia A and B are sex-linked recessive disorders with distinct deficiencies in factors 8 and 9, leading to significant bleeding issues.
- Recent advancements in gene therapy, such as Roktavian and Hemegenics, are promising for reducing bleeding rates in hemophilia patients.
Deep dives
Fundamentals of Hemophilia A and B
Hemophilia A is characterized by a congenital deficiency of factor 8, while hemophilia B is due to a deficiency of factor 9. Both conditions are sex-linked recessive disorders predominantly affecting males and are often diagnosed through a family history of bleeding. Clinical presentations include various types of bleeding, especially into weight-bearing joints, which can lead to hemophilic arthropathy. Diagnosis involves a normal PT and platelet count but a prolonged PTT, confirmed through specific factor assays that detail activity levels of factors 8 and 9.
