Updates, News in Sickle Cell Disease, with Steven Fein, MD

In part 4 of our 5-part series highlighting updates in hematology from 2024, Steven Fein, MD, a telehealth hematologist with HemeOnCall, discusses updates within sickle cell during the past year. Fein addresses setbacks and ongoing challenges in the hematology landscape for 2024. He notes the removal of voxelotor for sickle cell disease following findings that it was less effective than initially anticipated, leaving patients with fewer treatment options. While the approval of gene therapy for sickle cell disease in 2023 was a milestone, Fein believes routine implementation remains far off, emphasizing the need for better palliative treatments in the interim.

He also highlights the growing focus on access to care and equity in hematology, though progress has been slow. Fein points to issues like reduced access to chronic myeloid leukemia (CML) treatments as certain medications go generic, limiting patient assistance programs. Despite advances, he observes a lack of substantial improvement in access and coverage for patients, underscoring persistent barriers in equitable care delivery.

Relevant disclosures for Fein include Pharmacosmos Therapeutics, Sobi, Amgen, Agios, BeiGene, and others.