VJHemOnc Podcast
Key updates in sickle cell disease at ASH 2023: disease biology, ongoing trials & the growing role of gene therapy
Jan 12, 2024
Leading experts Rabi Hanna, MD and Josu de la Fuente, PhD discuss key updates in sickle cell disease at ASH 2023. They cover topics such as disease biology, ongoing trials, and the growing role of gene therapy. They explore novel agents, pathogenesis, and potential therapies, highlighting improvements in natural history, mortality rates, and access to healthcare. The speakers also discuss advancements in treatment, including the reduction in vasoclecive events and hospitalizations, and the paradigm shift towards increasing fetal hemoglobin for management.
11:05
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Quick takeaways
- The 2023 ASH Annual Meeting highlighted the improvement in the natural history of sickle cell disease, with increased median life expectancy and decreased mortality for pediatric and adult patients, emphasizing the need for better access to healthcare for adult hematologists to further improve patient outcomes.
- Exciting advancements in sickle cell disease treatment were discussed at ASH 2023, including positive results for a perovate kinase activator that improves hemoglobin levels and decreases hospitalizations, as well as the FDA approval of two gene therapy agents that offer new hope with reduced vaso-occlusive events and hospitalizations.
Deep dives
Improvement in Natural History of Sickle Cell Disease
At the 2023 Ash annual meeting, a study presented by the University of Cincinnati highlighted the improvement in the natural history of sickle cell disease over the last four decades. The median life expectancy has increased from 39 years to 43 years, with a significant decrease in mortality for pediatric and adult patients. However, there is still a need for better access to healthcare for adult hematologists to improve patient outcomes.
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