靶向 BCMA 的 CAR-T 细胞治疗浆细胞白血病的安全性和疗效

Despite significant therapeutic advances in multiple myeloma (MM), outcomes in patients with plasma cell leukemia (PCL) remain dismal.

We conducted a multicenter retrospective analysis of patients with PCL who were treated with the B-cell maturation antigen-directed chimeric antigen receptor T-cell (CAR-T) products idecabtagene vicleucel (ide-cel) and ciltacabtagene autoleucel (cilta-cel).

We identified 34 patients; 19 patients received ide-cel and 15 received cilta-cel. With a median follow-up of 11. 9 months, the overall median progression-free survival (mPFS) was 9. 0 months (95% confidence interval [CI], 4-15) and the median overall survival (mOS) was 13. 0 months (95% CI, 8 to not estimable [NE]). The 1-year cumulative incidence of progression or death was 72%, and the 1-year cumulative incidence of death was 47%. Patients who received cilta-cel had a longer mPFS (19. 0 months vs 6. 0 months) and mOS (>23 months [NE] vs 9. 0 months) when compared with those treated with ide-cel.

Similarly, the 1-year cumulative incidence of disease progression or death was 37. 5% (95% CI, 17. 4-68. 5) with cilta-cel, whereas all patients treated with ide-cel progressed or died within 12 months of infusion. The rates of hematologic and nonhematologic toxicities were similar between patients treated with cilta-cel and those treated with ide-cel and were consistent with those reported in patients with MM.

In this first multicenter study that evaluated patients with PCL who were treated with standard-of-care CAR-T products, we show that CAR-T is safe, feasible, and associated with improved outcomes when compared with historic standards.