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VJHemOnc Podcast

Post-ASH non-malignant highlights: updates in sickle cell disease, thalassemia, and hemophilia

Feb 14, 2025
Dr. Rabi Hanna from Cleveland Clinic shares insights on innovative treatments for severe sickle cell disease. He presents updated results from the RUBY trial, highlighting the potential of reni-cel as a groundbreaking gene therapy. The conversation also touches on the promising outcomes of the HIBISCUS trial for Etavopivat, showing significant improvements in patient quality of life. Furthermore, advancements in thalassemia treatments are discussed, focusing on reduced transfusion needs from the ENERGIZE-T study.
18:28

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Quick takeaways

  • The HIBISCUS trial demonstrates that Etavopivat significantly reduces painful vaso-occlusive crises in sickle cell disease patients, enhancing their quality of life.
  • The Energize-T trial shows that Metapivat effectively reduces transfusion needs in transfusion-dependent thalassemia patients, with a reassuring safety profile.

Deep dives

Updates on Sickle Cell Disease Treatments

The podcast highlights significant advancements in treatments for sickle cell disease, focusing on the hibiscus trial results involving Etavopivat, a pyruvate kinase activator. This Phase 2 trial, the largest of its kind, reported a 46% reduction in painful vaso-occlusive crises over a year and improvements in hemoglobin levels among participants. Patients receiving Etavopivat also experienced fewer crises compared to the placebo group, along with notable enhancements in quality of life indicators such as fatigue scores. Safety observations indicated that the drug was well tolerated, with adverse events being mild to moderate and resolving without significant medical interventions.

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