Due to concerns about poor response and treatment-related neurotoxicity, few studies have documented chimeric antigen receptor (CAR) T-cell therapy for patients with B-cell acute lymphoblastic leukemia (B-ALL) with central nervous system leukemia (CNSL). 

For a study, researchers sought to assess the effectiveness and safety of CD19-specific CAR T cell-based treatment; they enrolled 48 patients with relapsed/refractory B-ALL with CNSL in their research. In bone marrow (BM) illness, the infusion resulted in an overall response rate of 87.5% (95% CI, 75.3-94.1) and a remission rate of 85.4% (95% CI, 72.8-92.8) in CNSL. With a median follow-up of 11.5 months (range, 1.3-33.3), event-free survival was 8.7 months (95% CI, 3.7-18.8), and overall survival was 16.0 months (95% CI, 13.5-20.1). Due to concerns about poor response and treatment-related neurotoxicity, few studies have documented chimeric antigen receptor (CAR) T-cell therapy for patients with B-cell acute lymphoblastic leukemia (B-ALL) with central nervous system leukemia (CNSL).

At 12 months, the cumulative incidences of recurrence in BM and CNS disorders were 31.1% and 11.3%, respectively (P=.040). The medication was generally well-tolerated, with just 9 patients (18.8%) developing grade ≥3cytokine release syndrome. Grade 3 to 4 neurotoxic episodes, which occurred in 11 (22.9%) of the patients, were related to a larger preinfusion disease load in the CNS and were well managed under rigorous care. The findings implied that CD19-specific CAR T cell-based treatment could achieve comparable high response rates in both BM and CNS disorders. In CNSL, the length of remission was longer than in BM illness. CD19 CAR T-cell therapy with controllable neurotoxicity may give a possible therapeutic option for previously excluded individuals with CNSL.

Reference: ashpublications.org/blood/article/139/23/3376/484499/Efficacy-and-safety-of-CD19-specific-CAR-T-cell