自体干细胞移植、CAR-T 细胞治疗或联合巩固治疗用于再次获得完全缓解的复发/难治性原发性中枢神经系统淋巴瘤：一项逆概率治疗加权调整的对比研究

Chimeric antigen receptor T cell (CAR-T) therapy has emerged as a promising strategy for treating relapsed or refractory (R/R) primary central nervous system lymphoma (PCNSL). While recent reports suggest that combined autologous stem cell transplantation (ASCT) and CAR-T therapy may offer superior efficacy over CAR-T monotherapy, direct comparative evidence evaluating these modalities against conventional ASCT as consolidation therapy for patients in complete remission (CR) remains scarce.

This study was conducted to evaluate and compare the clinical efficacy and survival outcomes of 3 consolidation strategies-ASCT, CAR-T monotherapy, and ASCT combined with CAR-T therapy (ASCT+CAR-T)-in R/R PCNSL patients who reachieved CR. This retrospective study analyzed 112 patients with R/R PCNSL who received consolidation therapy at Beijing GoBroad Hospital between April 2022 and November 2025. CAR-T therapy recipients were enrolled in the clinical trial (ChiCTR2200058972). The median age of the cohort was 57 years (range, 25 to 75 years). Fourteen patients (12. 5%) had received more than 3 prior lines of therapy, and 23. 2% of the cohort presented with an International Extranodal Lymphoma Study Group (IESLG) score 3. For consolidation, 38 patients received CAR-T therapy, 42 underwent ASCT, and 32 received combined ASCT+CAR-T therapy. At a median follow-up of 21. 4 months, the overall 2-year progression-free survival (PFS) and overall survival (OS) rates were 70.

7% and 82. 5%, respectively. In the stabilized inverse probability of treatment weighting-adjusted analysis, the ASCT group demonstrated a significantly lower cumulative incidence of relapse compared to the CAR-T group (subdistribution hazard ratio [sHR], 0. 236; P = . 034) and the ASCT+CAR-T group (sHR, 0. 121; P = . 009). While ASCT showed a favorable trend in prolonging PFS over CAR-T therapy (hazard ratio, 0. 384; P = . 072), no significant differences were observed in OS or 3-month complete response rate across the 3 cohorts.

Safety was manageable. The most common causes of death were disease progression (58. 8%) and infection (41. 2%). For R/R PCNSL patients who successfully reachieved CR, ASCT monotherapy was associated with a lower cumulative incidence of relapse compared with CAR-T monotherapy or ASCT+CAR-T therapy. Given the retrospective nature of this study and potential residual confounding, these findings require further prospective validation.