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.
Bijal D Shah, Chibuzo Iloabuchi, Sean Harrigan, Markqayne Ray, Aida Santaolalla Revenga, David Cobley, Francis Nissen, Steve Kanters, Olalekan O Oluwole
{"title":"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.","authors":"Bijal D Shah, Chibuzo Iloabuchi, Sean Harrigan, Markqayne Ray, Aida Santaolalla Revenga, David Cobley, Francis Nissen, Steve Kanters, Olalekan O Oluwole","doi":"10.18553/jmcp.2026.32.10.1296","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Objective: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":16170,"journal":{"name":"Journal of managed care & specialty pharmacy","volume":"32 10","pages":"1296-1310"},"PeriodicalIF":3.3000,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13621901/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of managed care & specialty pharmacy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.18553/jmcp.2026.32.10.1296","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
引用次数: 0
Abstract
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.
期刊介绍：
JMCP welcomes research studies conducted outside of the United States that are relevant to our readership. Our audience is primarily concerned with designing policies of formulary coverage, health benefit design, and pharmaceutical programs that are based on evidence from large populations of people. Studies of pharmacist interventions conducted outside the United States that have already been extensively studied within the United States and studies of small sample sizes in non-managed care environments outside of the United States (e.g., hospitals or community pharmacies) are generally of low interest to our readership. However, studies of health outcomes and costs assessed in large populations that provide evidence for formulary coverage, health benefit design, and pharmaceutical programs are of high interest to JMCP’s readership.
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号
