Efficacy and Safety of CAR-T Cell Therapy in Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia With Central Nervous System Involvement
In brief
CAR-T therapy achieves 82% remission in B-ALL patients with CNS disease, matching those without CNS involvement
In a retrospective series of 113 relapsed/refractory B-cell ALL patients, 22 with CNS leukemia attained an overall complete remission rate of 81.8% at day 28, identical to the 91 CNS-negative patients, and experienced similar rates of cytokine release syndrome and neurotoxicity. However, CNS-positive patients relapsed more often, especially in the CNS, and only those who received subsequent allogeneic stem-cell transplant showed improved long-term survival.
- Journal
- Cancer medicine (Q1)
- Published
- 1 September 2026
- Study design
- Cohort / observational study
- Evidence level
- Level 3, Low (CEBM 3b)
- Authors
- Yanghong Yu, Hui Xu, Lei Xue, Xingbing Wang
- PMID
- 42702862
- DOI
- 10.1002/cam4.72262
Why clinicians should know about it
- Picked for Hematology (paper of the day, 11 September 2026): CAR-T cell therapy efficacy and safety in B-ALL with CNS
Abstract
Central nervous system (CNS) involvement in B-cell acute lymphoblastic leukemia (B-ALL) is associated with relapse, treatment refractoriness, and poor prognosis. Although chimeric antigen receptor (CAR) T cell therapy has demonstrated remarkable efficacy in relapsed/refractory (R/R) B-ALL, its efficacy and safety in CNS leukemia (CNSL) remain unclear. We retrospectively analyzed 113 R/R B-ALL patients who received CAR-T cell therapy, stratifying them into CNS-positive (n = 22) and CNS-negative (n = 91) groups based on the presence of CNSL prior to infusion. At Day 28 after CAR-T infusion, the overall complete remission (CR) rate was 81.8%, with no significant difference between groups. The incidence of cytokine release syndrome (CRS) and neurotoxicity was comparable. The 3-year cumulative incidence of relapse (CIR), event-free survival (EFS), and overall survival (OS) were similar between groups. However, CNSL patients exhibit a higher cumulative relapse rate following CAR-T-induced remission, with an increased risk of CNS relapse compared to patients without CNS involvement. Multivariate analysis identified allo-HSCT as consolidation post-CAR-T as an independent protective factor for improved EFS and OS in CNSL patients. In conclusion, CAR-T therapy offers similar efficacy and safety in R/R B-ALL patients regardless of CNS involvement. Furthermore, CAR-T cell therapy was not sufficient to maintain sustained remission, and consolidative allo-HSCT may improve long-term survival in this high-risk group.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.