Ciltacabtagene Autoleucel in Relapsed or Refractory Multiple Myeloma

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In a Chinese phase II trial (CARTIFAN-1) reported in the Journal of Clinical Oncology, Mi et al found that the anti–B-cell maturation antigen chimeric antigen receptor (CAR) T-cell therapy ciltacabtagene autoleucel was active in patients with relapsed or refractory multiple myeloma.

Study Details

In the multicenter trial, 46 patients who had received three or more lines of prior therapy (including a proteasome inhibitor and immunomodulatory drug) received a single ciltacabtagene autoleucel infusion at a target dose of 0.75 × 106 CAR+ viable T cells/kg. The primary endpoint was overall response rate.


Median follow-up was 18 months. Partial response or better was achieved in 43 (89.6%, 95% confidence interval [CI] = 77.3%–96.5%) of 48 patients, with complete response or better in 37 patients (77.1%, 95% CI = 62.7%–88.0%). Median time to first response was 1 month.  

Median duration of response was not reached; 67.2% of responses were ongoing at ≥ 18 months. Median progression-free survival was not reached; rates at 12 and 18 months were 77.0% and 66.8%. The overall survival rate at 18 months was 78.7%.

Adverse Events

Grade 3 or 4 hematologic adverse events included neutropenia in 97.9% of patients, leukopenia in 93.8%, lymphopenia in 91.7%, thrombocytopenia in 54.2%, and anemia in 52.1%. Any-grade cytokine-release syndrome occurred in 97.9% of patients and was grade 3 or 4 in 35.4%. Infections occurred in 85.4% and were grade 3 or 4 in 37.5%. Treatment-related fatal adverse events occurred in eight patients (16.7%), with causes consisting of hemorrhage, sudden cardiac death, septic shock, hepatic failure, pneumonia, intracranial hemorrhage, multiple organ failure, and pulmonary mycosis.

The investigators concluded, “These data demonstrate a favorable risk-benefit profile for a single infusion of ciltacabtagene autoleucel, resulting in early, deep, and durable responses in heavily pretreated patients with relapsed or refractory multiple myeloma in China.”

Sai-Juan Chen, MD, PhD, of Shanghai Institute of Hematology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, is the corresponding author for the Journal of Clinical Oncology article.

Disclosure: The study was supported by Janssen Research & Development, LLC, Legend Biotech USA Inc, and others. For full disclosures of the study authors, visit

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