Vivien Sheehan, MD, PhD, on Choosing the Right Treatment Option for Patients With Sickle Cell Disease

“The big take home message to me is that we need to have the decision to go forward with a cell-based therapy be the patient and their family's choice and not be paternalistic and say ‘You must try every FDA-approved therapy for SCD first.’ Not only is that inappropriate, but their level of benefit, even with good response does not equal that of a cell-based therapy, nor does transfusion.”

Historically, the small molecule drug hydroxyurea has been the main treatment option for patients with sickle cell disease (SCD). This changed over the past 8 years however, and especially in late 2023 with the FDA’s simultaneous approval of Vertex Pharmaceuticals' and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel, marketed as Casgevy) and bluebird bio’s lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia). As such, patients with SCD are now faced with several options for the treatment of their disease, including 2 options that are based on an advanced therapeutic modality. In order to help patients make their decision, treating physicians and other concerned healthcare professionals need to evaluate each patients’ individual needs and disease case.

Vivien Sheehan, MD, PhD, an associate professor of pediatrics at Emory University, gave a talk related to this topic entitled “Current and emerging pharmacological therapies for sickle cell disease” at the 2024 Tandem Meetings |Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held in San Antonio, Texas, February 21-24, 2024. After her presentation, CGTLive® asked Sheehan about the key points of her talk and the main takeaway for the healthcare community. Sheehan emphasized that providers are faced with the choice to either take a paternalistic approach in deciding patients’ treatment for them or to enable patients and their families to choose their own treatment path. Sheehan argued in favor of the latter option, noting that methods like matching patients’ symptoms to clinical end points and evaluating patients’ particular pathophysiology may help with the decision-making process.

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REFERENCES
1. Sheehan V. Current and emerging pharmacological therapies for sickle cell disease. Presented at: 2024 Tandem Meetings, February 21-24, San Antonio, Texas.