Ciltacabtagene autoleucel (cilta-cel) is a chimeric antigen receptor (CAR) T-cell therapy targeting the B-cell maturation antigen (BCMA) approved for use in heavily pretreated patients with relapsed or refractory multiple myeloma (MM). In the open-label, phase 3 CARTITUDE-4 trial, researchers examined the effectiveness of cilta-cel as an earlier treatment line in patients refractory to lenalidomide.
In the report published in The New England Journal of Medicine, the authors stated that a single infusion of cilta-cel yielded a lower risk of disease progression or death than standard of care.
The phase 3 trial randomized 419 patients to either cilta-cel (n=208) or the physician’s choice of effective standard care (n=211). All participants had received 1-3 prior lines of therapy.
Cilta-Cel Improved Survival in Refractory MM
Over a median follow-up of 15.9 months (range, 0.1-27.3), median progression-free survival (PFS) was not reached in the cilta-cel group and was 11.8 months in the standard of care group (hazard ratio [HR], 0.26; 95% CI, 0.18-0.38; P<.001). The 12-month rates of PFS were 75.9% (95% CI, 69.4-81.1) and 48.6% (95% CI, 41.5-55.3) for cilta-cel and standard care, respectively.
Compared with the standard-care group, more patients in the cilta-cel group had an overall response (84.6% vs 67.3%), a complete response or better (73.1% vs 21.8%), and an absence of minimal residual disease (60.6% vs 15.6%). Death from any cause occurred in 39 patients in the cilta-cel group and 46 in the standard care group (HR, 0.78; 95% CI, 0.5-1.2).
In 176 patients who received cilta-cel in the as-treated population, 134 (76.1%) had cytokine release syndrome, 8 (4.5%) had immune effector cell-associated neurotoxicity syndrome, 1 had movement and neurocognitive symptoms, 16 (9.1%) had cranial nerve palsy, and 5 (2.8%) had CART-T-related peripheral neuropathy. The authors noted that most patients reported grade 3 or 4 adverse events during treatment.
Overall, the authors suggested that cilta-cel may be effective in earlier lines of treatment among patients with refractory MM.