Multinational phase 3 trial of ciltacabtagene autoleucel versus standard care for lenalidomide-refractory multiple myeloma

Ciltacabtagene autoleucel (cilta-cel), a BCMA-directed CAR-T cell therapy, is effective in heavily pretreated patients with relapsed or refractory multiple myeloma. The multinational phase 3 CARTITUDE-4 trial evaluated cilta-cel in earlier treatment lines in patients with lenalidomide-refractory disease. Patients with one to three previous lines of treatment were randomized to cilta-cel or physician’s choice of standard care. The median progression-free survival was not reached with cilta-cel and was 11.8 months with standard care (HR 0.26; p<0.001). Overall response, complete response or better, and MRD-negative disease were more frequent with cilta-cel. Most patients reported grade 3 or 4 adverse events with cilta-cel.