The real-world results of chimeric antigen receptor T-cell (CAR-T) therapy for patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL) substantially differ across countries. In the Netherlands, the CAR-T tumorboard facilitates a unique nationwide infrastructure for referral, eligibility assessment and data collection. The aim of this study was to evaluate real-world outcomes of axicabtagene ciloleucel (axi-cel) in the Dutch population, including the thus-far underreported effects on health-related quality of life (HR-QoL). All patients with R/R LBCL after ≥2 lines of systemic therapy referred for axi-cel treatment between May 2020–May 2022 were included (N = 250). Of the 160 apheresed patients, 145 patients received an axi-cel infusion. The main reason for ineligibility was rapidly progressive disease. The outcomes are better or at least comparable to other studies (best overall response rate: 84% (complete response: 66%); 12-month progression-free-survival rate and overall survival rate: 48% and 62%, respectively). The 12-month NRM was 5%, mainly caused by infections. Clinically meaningful improvement in several HR-QoL domains was observed from Month 9 onwards. Expert-directed patient selection can support effective and sustainable application of CAR-T treatment. Matched comparisons between cohorts will help to understand the differences in outcomes across countries and select best practices. Despite the favorable results, for a considerable proportion of patients with R/R LBCL there still is an unmet medical need.
嵌合抗原受体T细胞（CAR-T）疗法在复发/难治性大B细胞淋巴瘤患者中的真实世界疗效在不同国家间存在显著差异。在荷兰，CAR-T肿瘤委员会建立了全国性的转诊、资格评估和数据收集体系。本研究旨在评估阿基仑赛在荷兰人群中的真实世界疗效，包括目前报道较少的健康相关生活质量影响。研究纳入了2020年5月至2022年5月期间接受≥2线系统治疗后转诊接受阿基仑赛治疗的所有复发/难治性大B细胞淋巴瘤患者（N=250）。在160例完成单采的患者中，145例接受了阿基仑赛输注。主要排除原因为疾病快速进展。治疗结果优于或至少与其他研究相当（最佳总缓解率：84%（完全缓解率：66%）；12个月无进展生存率和总生存率分别为48%和62%）。12个月非复发死亡率为5%，主要死因为感染。从第9个月开始，多个健康相关生活质量领域出现具有临床意义的改善。专家指导的患者筛选有助于CAR-T治疗的有效和可持续应用。队列间的匹配比较将有助于理解各国疗效差异并选择最佳实践。尽管结果良好，但对于相当比例的复发/难治性大B细胞淋巴瘤患者，仍存在未满足的医疗需求。
肿瘤(癌症)患者之家