不适合移植的大 B 细胞淋巴瘤二线使用 axicabtagene ciloleucel:ALYCANTE 最终分析
Second-line axicabtagene ciloleucel in large B-cell lymphoma ineligible for transplantation: ALYCANTE final analysis.
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Retrospective Cohort Analysis of Treatment Patterns, Survival, and Cost-Effectiveness in Relapsed/Refractory Diffuse Large B-Cell Lymphoma in Lower Austria (2018-2022).
Retrospective Cohort Analysis of Treatment Patterns, Survival, and Cost-Effectiveness in Relapsed/Refractory Diffuse Large B-Cell Lymphoma in Lower Austria (2018-2022).
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r/r DLBCL 的现代疗法显著提高了生存率,但其高昂成本需要审慎的成本效果评估,以确保其以最优方式融入临床实践。
复发/难治性(R/R)弥漫性大B细胞淋巴瘤(DLBCL)是一种侵袭性恶性肿瘤,治疗选择有限。新型免疫疗法改善了特定临床试验人群的结局,但其在真实世界中的有效性仍不明确,且高昂费用也给卫生系统带来挑战。
本探索性研究在真实世界人群中评估现有疗法的疗效和费用。采用两种回顾性分析方法。首先比较按日历时间划分的队列:常规治疗时期(2018年1月1日至2019年12月31日,早于欧洲药品管理局批准泊洛妥珠单抗维多汀)与现代治疗时期(2020年1月1日至2022年12月31日)。其次,由于不同治疗时期存在方案重叠,进一步按实际治疗暴露分组,比较任一阶段接受过现代疗法者与仅接受常规方案者。
按日历时期比较所得信息有限,因为早期尚不可用的疗法仍可能在后续治疗线中使用。相比之下,按任一阶段是否接受现代疗法分组后,发现明确的生存获益:中位总生存期(OS)为20个月对5个月(95% CI:1.8–38.2个月对3.0–7.0个月;p=0.022)。这一优势伴随平均费用显著升高(178,513.08对15,185.08;p<0.001),每增加一个月生存期的增量费用为10,889。
R/R DLBCL现代疗法显著提高了生存率,但其高昂费用要求开展审慎的成本效益评估,以确保在临床实践中得到最佳整合。此外,我们的发现提示,按日历时期比较可能产生误导,因为新疗法并非在获批日期简单地“开始”或“停止”使用——这是对真实世界证据研究具有普遍意义的重要方法学启示。按实际治疗暴露分组,在新疗法成为常规治疗后,可更准确地评估其临床获益和经济影响。要在维持医疗体系可持续性的同时实现公平治疗可及,需要临床医生、研究者和政策制定者协同努力。
R/R DLBCL is an aggressive malignancy with limited treatment options. Novel immunotherapies have improved outcomes in selected clinical-trial populations, but their effectiveness in real-world settings remains unclear. Their substantial costs also pose challenges for healthcare systems.
This exploratory study evaluated the efficacy and costs of available therapies in a real-world population. Two retrospective analysis approaches were used: First, we compared calendar-based cohorts: A conventional-treatment period (1 January 2018-31 December 2019), which predates EMA approval of Polatuzumab-vedotin, and a modern-treatment period (1 January 2020-31 December 2022). Second, because treatments overlapped across periods, we additionally grouped patients by therapy exposure and compared those receiving any modern therapy at any stage with those treated exclusively with conventional regimens.
Comparing the calendar-based periods provided limited insights, as therapies unavailable in the earlier period could still be used in subsequent treatment lines. In contrast, grouping patients by exposure to modern therapy at any stage showed a clear survival benefit: Median OS was 20 vs. 5 months (95% CI: 1.8-38.2 vs. 3.0-7.0; p = 0.022). This advantage was associated with significantly higher mean costs ( 178,513.08 vs. 15,185.08; p < 0.001), resulting in an incremental cost of 10,889 per additional month of survival.
Modern therapies for r/r DLBCL have significantly improved survival rates, but their high costs necessitate careful cost-effectiveness assessments to ensure they are integrated optimally into clinical practice. Additionally, our findings indicate that calendar-based comparisons can be misleading, since novel treatments do not simply "start" and "stop" at approval dates-a crucial methodological insight that is highly generalizable to real-world evidence studies. Grouping patients by actual therapy exposure offers a more accurate evaluation of clinical benefits and economic impact when new treatments become routine. Achieving equitable access to treatment while maintaining healthcare sustainability will require coordinated efforts among clinicians, researchers, and policymakers.
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