Journal: Blood cancer journal
This publication reports real-world outcomes for ciltacabtagene autoleucel (cilta-cel), an anti-BCMA CAR-T therapy, in relapsed/refractory multiple myeloma using Center for International Blood and Marrow Transplant Research registry data (March 2022–December 2023).
Study population:
- 595 treated patients; median age 64; 57% male.
- High comorbidity burden: 70% with ≥1 comorbidity.
- High-risk disease features: extramedullary disease in 13%; bone marrow plasma cells ≥50% in 14%.
- Heavily pretreated: median 7 prior lines; 8% with prior BCMA-directed therapy.
- Median follow-up: 12 months (range 1–25).
Safety:
- Cytokine release syndrome: 80%; grade ≥3 in 4%.
- ICANS: 22%; grade ≥3 in 4%.
- Non-ICANS neurotoxicity: 5% (31 patients), including:
- Parkinsonism: 2.7% (16 patients).
- Cranial nerve palsies: 2.5% (15 patients), mainly facial nerve (cranial nerve VII; 12/15).
- Infections: 47%.
- Treatment-related mortality: 5%.
Efficacy:
- Best overall response rate: 87%.
- ≥ very good partial response: 75%.
- ≥ complete response: 35%.
- Estimated 12-month progression-free survival: 73% (95% CI 68–77%).
- Estimated 12-month overall survival: 85% (95% CI 81–88%).
In summary, in the largest standard-of-care series to date, cilta-cel showed high response rates and encouraging 1-year PFS/OS in a heavily pretreated, comorbid RRMM population, with a safety profile consistent with CAR-T therapy and notable but relatively infrequent serious neurologic events and treatment-related mortality.