From the USA: Exa-Cel Eliminates Vaso-Occlusive Crises in Most Sickle Cell Patients
For almost all patients with sickle cell disease, treatment with exagamglogene autotemcel (exa-cel) eliminates vaso-occlusive crises, according to a study published in the April 24 issue of the New England Journal of Medicine.
Haydar Frangoul, M.D., from the Sarah Cannon Research Institute at the Children’s Hospital at TriStar Centennial in Nashville, Tennessee, and colleagues conducted a phase 3, open-label study of exa-cel in patients aged 12 to 35 years of age with sickle cell disease with at least two severe vaso-occlusive crises in each of the two years before screening. Patients underwent myeloablative conditioning with pharmacokinetically dose-adjusted busulfan before exa-cel infusion. Forty-four patients received exa-cel and were followed for a median of 19.3 months.
Source: Advances and More licensed by HealthDay
