Abstract / Summary
Background: Brexucabtagene autoleucel (brexu-cel), a CD19 chimeric antigen receptor T-cell therapy, has shown efficacy in clinical trials for relapsed/refractory (R/R) mantle cell lymphoma (MCL) and B-cell acute lymphoblastic leukemia (B-ALL). However, although effectiveness and safety evidence is well established, real-world evidence (RWE) on health care resource use (HCRU), costs, and health-related quality of life (HRQoL) remains limited.
Objective: To systematically review and synthesize RWE on clinical, HCRU, and cost outcomes, and both RWE and trial evidence on HRQoL, for brexu-cel in adults with R/R MCL and B-ALL.
Methods: Systematic searches were conducted to identify studies reporting outcomes for adults with R/R MCL and B-ALL treated with brexu-cel. RWE was included for clinical, HCRU, and costs outcomes, and HRQoL outcomes. Effectiveness and safety were analyzed using meta-analysis, while HCRU, cost, and HRQoL were summarized descriptively.
Results: 69 publications representing 31 cohorts were identified (19 MCL, 7 B-ALL, 3 mixed). Pooled overall response and complete response rates in MCL were 88% and 76%, while B-ALL showed complete response and measurable residual disease negativity of 74% and 73%, consistent with pivotal trials ZUMA-2 and ZUMA-3, respectively. Median overall survival was 40.3 months in MCL and 23.3 months in B-ALL. Cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome were the most common adverse events; severe events were less frequent in real-world cohorts compared with trials, potentially reflecting advances in safety management and earlier identification of toxicities. HCRU and cost evidence was limited; 1 MCL study reported reduced post-chimeric antigen receptor T costs. No real-world HRQoL data were identified.
Conclusions: Brexu-cel provides substantial and durable benefit in R/R MCL and B-ALL, with real-world effectiveness and safety consistent with pivotal trials. However, evidence gaps remain for HRQoL and economic outcomes, underscoring priorities for future research.