Multicenter real-world study of ciltacabtagene autoleucel for relapsed or refractory multiple myeloma

Ciltacabtagene autoleucel (cilta-cel) was approved in 2022 for patients with relapsed or refractory multiple myeloma (RRMM) based on results of the phase 2 CARTITUDE-1 trial. A study at 16 US academic centers evaluated cilta-cel for RRMM in the real world. Among the 255 patients who underwent leukapheresis, 236 (92.5%) received cilta-cel. Median prior lines of therapy were 6. CRS was seen in 75% (grade 3 or higher in 5%), ICANS in 14% (grade 3 or higher in 4%), and delayed neurotoxicity in 10%. Overall response was seen in 89% and complete response or better in 70%. Nonrelapse mortality was 10%, most commonly from infection. After median follow-up of 13 months from cilta-cel infusion, median progression-free survival was not reached, with 12-months PFS rate 68% (95% CI 62-74). Second primary malignancies excluding non-melanoma skin cancers were seen in 5.5% and myeloid malignancies/AML in 1.7%. The authors conclude that cilta-cel for RRMM in clinical practice had a favorable efficacy profile.