Efficacy of BCMA CAR-T cell therapy and subsequent strategies in refractory and relapsed plasma cell leukemia: a retrospective cohort study.
Plasma cell leukemia (PCL) is a rare and aggressive hematological malignancy. The long-term prognosis of relapsed/refractory plasma cell leukemia (R/R PCL) remains poor, and few treatment options are available for patients with triple-refractory disease. Chimeric antigen receptor (CAR)-T cell therapy targeting the B-cell maturation antigen (BCMA) has shown promise, though its long-term efficacy and optimal subsequent strategies remain to be fully elucidated.
This retrospective study analyzed the efficacy and safety of BCMA CAR-T therapy in 12 patients with triple-class R/R PCL. Patients were stratified into consolidation (Group 1, allo-HSCT within 3 months post-CAR-T) and non-consolidation (Group 2, no allo-HSCT within 3 months post-CAR-T) groups, with survival outcomes compared between cohorts.
The overall response rate following BCMA-CAR-T cell therapy was 75% (9/12); four patients achieved partial response, four achieved very good partial response, and one patient had complete response. Grade 3-4 cytopenia were universally observed, while 83.3% (10/12)of the patients presented with mild (grade 1-2) cytokine release syndrome. The median progression free survival (PFS) was 8.9 months (95% CI: 4.6, not reached). The 1-year PFS rate was 33.3% (95% CI: 7.8-62.3), and the 2-year PFS rate was 22.2% (95% CI: 3.4-51.3). The median overall survival (OS) was 15.5 months (95% CI: 5.7, not reached). The 1-year OS rate was 55.6% (95% CI: 20.4-80.5), and the 2-year OS rate was 22.2% (95% CI: 3.4-51.3). furthermore, two of the four patients who underwent consolidation therapy showed long-term survival with stringent complete response.
BCMA-CAR-T therapy confers short-term remission and survival benefits in relapsed/refractory plasma cell leukemia (R/R PCL). However, the definitive value of allogeneic hematopoietic stem cell transplantation (allo-HSCT) awaits validation in large-sample prospective studies.
This retrospective study analyzed the efficacy and safety of BCMA CAR-T therapy in 12 patients with triple-class R/R PCL. Patients were stratified into consolidation (Group 1, allo-HSCT within 3 months post-CAR-T) and non-consolidation (Group 2, no allo-HSCT within 3 months post-CAR-T) groups, with survival outcomes compared between cohorts.
The overall response rate following BCMA-CAR-T cell therapy was 75% (9/12); four patients achieved partial response, four achieved very good partial response, and one patient had complete response. Grade 3-4 cytopenia were universally observed, while 83.3% (10/12)of the patients presented with mild (grade 1-2) cytokine release syndrome. The median progression free survival (PFS) was 8.9 months (95% CI: 4.6, not reached). The 1-year PFS rate was 33.3% (95% CI: 7.8-62.3), and the 2-year PFS rate was 22.2% (95% CI: 3.4-51.3). The median overall survival (OS) was 15.5 months (95% CI: 5.7, not reached). The 1-year OS rate was 55.6% (95% CI: 20.4-80.5), and the 2-year OS rate was 22.2% (95% CI: 3.4-51.3). furthermore, two of the four patients who underwent consolidation therapy showed long-term survival with stringent complete response.
BCMA-CAR-T therapy confers short-term remission and survival benefits in relapsed/refractory plasma cell leukemia (R/R PCL). However, the definitive value of allogeneic hematopoietic stem cell transplantation (allo-HSCT) awaits validation in large-sample prospective studies.
Authors
Guo Guo, Ma Ma, Yang Yang, Fu Fu, Li Li, Liu Liu, Cao Cao, Wei Wei, Dou Dou, Deng Deng, Gan Gan, Chang Chang, Ke Ke, Hu Hu
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