By Patrick Daly Last Updated: November 29, 2023
Compared with effective standard-of-care therapies, ciltacabtagene autoleucel (cilta-cel) improved survival outcomes in pretreated patients with lenalidomide-refractory multiple myeloma, according to a clinical trial published in the New England Journal of Medicine.
The open-label, phase III, randomized, controlled trial was led by Jesús San-Miguel, MD, PhD, of the Cancer Center Clínica Universidad de Navarra in Pamplona, Spain. Investigators randomized patients to cilta-cel or the treating physician’s choice of effective care.
Overall, 419 patients were enrolled and assigned to either cilta-cel (n=208) or standard care (n=211). At a median follow-up of 15.9 months (range, 0.1-27.3 months), the median progression-free survival (PFS) was not reached with cilta-cel versus 11.8 months with standard care (hazard ratio [HR], 0.26; 95% CI, 0.18-0.38; P<.001).
The 12-month PFS rate was 75.9% (95% CI, 69.4-81.1) in the cilta-cel group versus 48.6% (95% CI, 41.5-55.3) in the standard care group. Additionally, more patients in the cilta-cel group had an overall response, complete response or better, and measurable residual disease negativity at 84.6%, 63.1%, and 60.6%, respectively, compared with standard care at 67.3%, 21.8%, and 15.6%, respectively.
Researchers reported most patients experienced grade 3 or 4 adverse events during treatment, and 39 patients in the cilta-cel group and 46 in the standard care group died (HR, 0.78; 95% CI, 0.5-1.2).
Among 176 as-treated patients in the cilta-cel group, 134 (76.1%) developed cytokine release syndrome, 16 (9.1%) had cranial nerve palsy, eight (4.5%) had immune effector cell-associated neurotoxicity syndrome, five (2.8%) had chimeric antigen receptor T-cell-related peripheral neuropathy, and one had movement and neurocognitive symptoms.
Ultimately, “a single cilta-cel infusion resulted in a lower risk of disease progression or death than standard care in lenalidomide-refractory patients with multiple myeloma who had received one to three previous therapies,” Dr. San-Miguel and colleagues concluded.
Reference
San-Miguel J, Dhakal B, Yong K, et al. Cilta-cel or standard care in lenalidomide-refractory multiple myeloma. N Engl J Med. 2023;389(4):335-347. doi:10.1056/NEJMoa2303379
Original Source: Cilta-cel Improved Outcomes in Pretreated Multiple Myeloma Compared with Standard Care | Blood Cancers Today