Shalini Shenoy, MD, MBBS, on Comparing Treatment Options for Sickle Cell Disease
The director of the Stem Cell Transplant & Cellular Therapy Program at St. Louis Children’s Hospital discussed when the choice should be made to transition from symptom management to curative therapies.
“[Patients with SCD] should have a normal lifestyle [on drugs for symptom management], and if they don't then it's time to consider a curative therapy for these patients, whether they're children or adults.”
Currently, treatment options for sickle cell disease (SCD) mainly fall into 1 of 2 main categories. The first category is drugs that are mainly intended for symptom management and disease control. The second category is potentially curative treatment options. The latter group mainly consists of allogeneic hematopoietic stem cell transplantation (allo-HSCT) and gene therapies, 2 of which were recently approved by the FDA: Vertex Pharmaceuticals' and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel, marketed as Casgevy) and bluebird bio’s lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia). For some patients, drugs for symptom management are sufficient for a satisfactory quality of life with SCD, but other patients are refractory to these drugs or do not receive sufficient benefit from them. As such, patients in the latter group may wish to seek a curative therapy. Although curative therapies may be able to provide a transformative effect with a 1-time treatment for some patients, they carry some drawbacks, such as potential risks and high costs not seen with drugs for symptom management. Therefore, choosing the right treatment option for each patient with SCD requires careful consideration.
This complex and changing landscape of care for SCD was the topic of a session entitled “The evolving paradigm of sickle cell disease management” that was held at the 2024 Tandem Meetings |Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR, held in San Antonio, Texas, February 21-24, 2024. After the session, CGTLive® sat down with Shalini Shenoy, MD, MBBS, the director of the Stem Cell Transplant & Cellular Therapy Program at St. Louis Children’s Hospital, who spoke about allo-HSCT during the session, to get her perspective on what was discussed. Shenoy gave an overview of the key points and main takeaways from the session, noting that advancements in allo-HSCT technology and the recent gene therapy approvals are contributing to a changing of the standard of care for SCD.
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REFERENCES
1. Sharma A, Sheehan V, Shenoy S. Tandem Thursday breakfast symposium: the evolving paradigm of sickle cell disease management. Presented at: 2024 Tandem Meetings, February 21-24, San Antonio, Texas. Session #S-B1.
Considerations for Administering Hemoglobinopathy Gene Therapies From a Pharmacist’s Perspective
April 20th 2024Alexis Kuhn, PharmD, BCOP, a pediatric oncology pharmacist at Mayo Clinic, discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.
