Ciltacabtagene autoleucel (cilta-cel), a BCMA-targeting chimeric antigen receptor (CAR) T-cell therapy, may significantly slow or stop the progression of multiple myeloma (MM)  when compared with standard-of-care treatments among patients for whom lenalidomide no longer works, according to new data presented at the ASCO Annual Meeting 2023.

The global, phase 3 CARTITUDE-4 clinical trial ( Identifier: NCT04181827) included 419 patients with MM who had already received 1 to 3 lines of treatment, including lenalidomide, which was no longer effective.

There were 208 participants in the ciltacabtagene autoleucel arm and 211 participants in the standard-of-care treatment arm. Standard-of-care included either a combination of bortezomib, pomalidomide, and dexamethasone or a combination of daratumumab, pomalidomide, and dexamethasone.

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CARTITUDE-4 enrolled participants from 16 countries, including the United States, Europe, Asia, and Australia. The median age was 61 years, 55% were male, and 75% were White. The data were presented by lead study author Binod Dhakal, MD, with the Medical College of Wisconsin, Milwaukee, Wisconsin.

Cilta-cel reduced the risk of disease progression by 74% (hazard ratio [HR], 0.26; P <.0001). Compared with patients assigned to current standard-of-care regimens, the patients in the cilta-cel arm had significantly improved objective response rates (ORR; 84.6% vs 67.3%), rates of complete response (73.1% vs 22.8%), and overall rates of negative minimal residual disease (MRD; 87.5% vs 32.7%).

There was also a positive trend in overall survival (HR 0.78; 95% CI, 0.5-1.2), although the data were immature, with 39 deaths in the cilta-cel arm vs 47 deaths in the standard-of-care arm.  

Most patients (96.6% receiving cilta-cel and 94.2% receiving standard of care) experienced grade 3/4 adverse events. These included infections (26.9% with cilta-cel vs 24.5% standard of care) and low blood cell counts (94.2% vs 86.1%), including neutropenia, thrombocytopenia, and anemia.

Cytokine release syndrome developed in 76.1% of participants who received cilta-cel. Approximately 5% of those receiving the CAR-T therapy developed immune effector cell-associated neurotoxicity syndrome. Additional analyses of the data are ongoing, such as health-related quality of life, subgroup analyses, and biomarker analyses.

Disclosures: This research was supported by Janssen Research & Development, LLC. Some study authors declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of disclosures.


Dhakal B, Yong K, Harrison SJ, et al. First phase 3 results from CARTITUDE-4: cilta-cel versus standard of care (PVd or DPd) in lenalidomide-refractory multiple myeloma. ASCO 2023. June 2-6, 2023. Abstract LBA106.