Post-ASH non-malignant highlights: updates in sickle cell disease, thalassemia, and hemophilia
Feb 14, 2025
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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.
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.
Gene Therapy Innovations for Sickle Cell Disease
The RUBI trial is discussed, showcasing the use of RenniCell gene therapy which employs gene editing technology to treat severe sickle cell disease. An update from the trial revealed consistent safety profiles, with no reported fatalities and only two adverse effects stemming from conditioning therapy. The results demonstrated a robust clinical efficiency, with most patients showing significant improvement in pain crises, particularly noteworthy given that one patient had a history of multiple pain crises prior to enrollment. These findings suggest promising advancements in gene therapy, which, despite challenges in accessibility, could revolutionize treatment options for patients.
Energize-T Study Outcomes in Thalassemia
The podcast provides insights into the Energize-T trial focused on the efficacy of Metapivat for transfusion-dependent thalassemia patients, with the trial achieving significant success in reducing transfusion burdens. The study's primary endpoint was met, showing a statistically significant decline in transfusion needs for patients treated with Metapivat, with key secondary endpoints also reaching their goals. The safety profile appeared reassuring, as any adverse events reported were comparable to those in the placebo group and mostly transient. This research underscores the potential of Metapivat as a viable treatment alternative for managing thalassemia, paving the way for further approvals and studies.
This episode of the VJHemOnc podcast covers updates in non-malignant hematological diseases from the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, which took place in San Diego, CA. You will hear the latest updates in sickle cell disease (SCD), thalassemia, and hemophilia from leading experts.
First, Sophia Delicou, MD, Hippokration General Hospital, Athens, Greece, will provide an update on the HIBISCUS (NCT04624659) trial of etavopivat in SCD, and Rabi Hanna, MD, Cleveland Clinic, Cleveland, OH, will present updated results of the RUBY (NCT04853576) trial investigating reni-cel in severe SCD. This will be followed by an update of a trial being conducted in patients with transfusion-dependent thalassemia, the ENERGIZE-T study (NCT04770779), from Maria Domenica Cappellini, MD, University of Milan, Milan, Italy. Finally, you will hear the results of the explorer7 trial (NCT04083781) in hemophilia A and B from Amy Shapiro, MD, Indiana University Hemophilia Center, Indianapolis, IN.