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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Episode guests

Rabi Hanna, MD

Josu de la Fuente, PhD, FRCP, FRCPI, FRCPCH, FRCPath

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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.

Introduction

3min

Updates on Disease Biology, Ongoing Trials, and Gene Therapy in Sickle Cell Disease

2min

Advancements in sickle cell disease treatment: Trials and gene therapy

4min

Paradigm Shift in the Management of Sickle Cell Disease

2min

In recent years, the treatment landscape of sickle cell disease (SCD) has been revolutionized with the introduction of novel agents, including recently approved gene therapies. There are several ongoing trials evaluating the safety of these agents in patients, with promising results being demonstrated.

In this podcast, leading experts Rabi Hanna, MD, Cleveland Clinic, Cleveland, OH, and Josu de la Fuente, PhD, FRCP, FRCPI, FRCPCH, FRCPath, Imperial College London, London, UK, discuss key updates in SCD presented at the 2023 ASH Annual Meeting and Exposition. The experts cover several topics, including the pathogenesis of SCD, novel agents being explored, and the growing role of gene therapies.

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