文章：

改善阿基仑赛可及性对健康的影响：以西班牙为例

The Health Impacts of Better Access to Axicabtagene Ciloleucel: The Case of Spain

原文发布日期：30 July 2024

DOI: 10.3390/cancers16152712

类型: Article

开放获取: 是

英文摘要：

In this study, the health impacts of improving access to treatment with axicabtagene ciloleucel (axi-cel) was assessed in patients with relapsed/refractory diffuse large B-cell lymphoma after ≥2 lines of therapy in Spain. A partitioned survival mixture cure model was used to estimate the lifetime accumulated life years gained (LYG) and quality-adjusted life years (QALYs) per patient treated with axi-cel versus chemotherapy. Efficacy data were extracted from the ZUMA-1 trial for axi-cel and from the SCHOLAR-1 study for chemotherapy. In the base case, the incremental outcomes of axi-cel versus chemotherapy were evaluated in a cohort of 187 patients treated with CAR T-cell therapies, as reported by the “Spanish National Health System Plan for Advanced Therapies”, and in the alternative scenario in the full eligible population based on epidemiological estimates (n = 490). Taking those currently treated with axi-cel, compared with chemotherapy, axi-cel provided an additional 1341 LYGs and 1053 QALYs. However, when all eligible patients (n = 490) were treated, axi-cel provided an additional 3515 LYs and 2759 QALYs. Therefore, if all eligible patients were treated with axi-cel rather than those currently treated as per the registry (n = 187), there would have been an additional 303 patients treated, resulting in an additional 2173 LYGs and 1706 QALYs in total. The lack of access in Spain has led to a loss of a substantial number of LYGs and QALYs, and efforts should be made to improve access for all eligible patients.

摘要翻译： 

本研究评估了西班牙在≥2线治疗后复发/难治性弥漫性大B细胞淋巴瘤患者中，提高阿基仑赛（axi-cel）治疗可及性对健康的影响。采用分区生存混合治愈模型，估算接受axi-cel治疗与化疗患者相比，终生累积获得的生命年（LYG）和质量调整生命年（QALYs）。疗效数据来源于ZUMA-1试验（axi-cel）和SCHOLAR-1研究（化疗）。基础案例分析中，根据《西班牙国家卫生系统先进疗法计划》报告的187例接受CAR-T细胞治疗患者队列，评估axi-cel相较于化疗的增量效益；替代情景则基于流行病学估算的全部符合条件人群（n=490）进行分析。在当前接受axi-cel治疗的患者中，与化疗相比，axi-cel额外提供了1341个LYG和1053个QALYs。然而，当所有符合条件患者（n=490）均接受治疗时，axi-cel可额外提供3515个LYG和2759个QALYs。因此，若所有符合条件患者均能接受axi-cel治疗（而非当前登记治疗的187例），将新增303例治疗患者，总计额外获得2173个LYG和1706个QALYs。西班牙当前治疗可及性的不足已导致大量LYG和QALYs的损失，应着力提升所有符合条件患者的治疗可及性。

原文链接：

The Health Impacts of Better Access to Axicabtagene Ciloleucel: The Case of Spain