IN the treatment of relapsed/refractory multiple myeloma, CAR T-cell therapy using idecabtagene vicleucel (ide-cel) has demonstrated encouraging results in a large real-world U.S. patient cohort, according to new data from the CIBMTR registry.
This study, the largest real-world evaluation to date, tracked 821 patients treated with standard-of-care ide-cel, offering a broader and more inclusive perspective than controlled clinical trials. Unlike trial populations, this cohort had a median age of 66 years, with 31% of patients aged 70 or older, 15% identifying as Black, 7% as Hispanic, and 77% presenting with at least one significant co-morbidity.
Despite the complexity and severity of their disease—patients had received a median of seven prior lines of therapy, 17% had extramedullary disease, and over a quarter had high-risk cytogenetics, treatment outcomes were promising. The overall response rate was 73%, with 25% achieving complete response. Median progression-free survival reached 8.8 months.
The safety profile aligned with prior trial data. Cytokine release syndrome (CRS) occurred in 80% of patients, though only 3% experienced grade 3 or higher. Immune effector cell–associated neurotoxicity syndrome (ICANS) was observed in 28%, with 5% being grade 3 or higher. Importantly, no cases of Parkinsonism were reported.
Treatment-related mortality was recorded in 6% of patients, and 45% experienced clinically significant infections. Second primary malignancies were reported in 4% of the population, with 1% being myeloid malignancies.
These findings underscore the real-world viability of ide-cel, even in patients who would not have qualified for the pivotal KarMMa clinical trial due to age or comorbid conditions.
The results offer reassuring evidence to oncologists and hematologists that CAR-T therapy can be safely and effectively delivered in standard practice settings across diverse and medically complex patients—providing renewed hope for those with limited treatment options.
Reference: Sidana S et al. Standard-of-Care Idecabtagene Vicleucel for Relapsed/Refractory Multiple Myeloma: A CIBMTR Analysis. Blood. 2025. 8:blood.2024026216.
