Real-World Efficacy Outcomes of Ciltacabtagene Autoleucel in Relapsed Refractory Multiple Myeloma: A Comparative...

The CARTITUDE-1 Trial (C-1) led to the approval of ciltacabtagene autoleucel (cilta-cel), a BCMA-directed CAR-T cell therapy, for patients with relapsed or refractory multiple myeloma (RRMM) after ≥ 3 lines of therapy. However, the inclusion criteria may not fully represent patients in the real-world (RW).

Leveraging a national multicenter cohort of patients with RRMM, we conducted a retrospective analysis comparing 73 patients who received cilta-cel in the RW to 97 patients treated in C-1 by evaluating clinical characteristics, depth of response, survival outcomes, and toxicity.

Results showed that the RW baseline demographics such as age, proportion of racial minorities, gender, prior therapies, and disease characteristics, including high-risk cytogenetics, were broadly similar to the C-1 population. In the RW group, fewer patients had Eastern Cooperative Oncology Group 0 (16% vs. 40%, P < .001), more patients had extramedullary disease (29% vs. 13%, P = .02), and fewer were refractory to bortezomib (47% vs. 68%, P = .007) and anti-CD38 monoclonal antibody (88% vs. 97%, P = .03). Forty-five percent of RW patients did not meet the inclusion criteria for C-1. The overall response rate was lower in the RW group (88% vs. 97%, P = .015). The survival outcomes at 24-months including the progression-free survival (58% in RW vs. 62% in C1, P = .98) and overall survival (72% vs. 74%, P = .9) were similar. The safety profile of RW patients was better with fewer treatment-related adverse events.

Our study demonstrates the comparable efficacy of cilta-cel in patients with RRMM treated in a RW setting.