Clinical, economic, and quality-of-life outcomes of brexucabtagene autoleucel for the treatment of relapsed/refractory mantle cell lymphoma and B-cell acute lymphoblastic leukemia: A systematic literature review and meta-analysis.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Authors
Shah Shah, Iloabuchi Iloabuchi, Harrigan Harrigan, Ray Ray, Santaolalla Revenga Santaolalla Revenga, Cobley Cobley, Nissen Nissen, Kanters Kanters, Oluwole Oluwole
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