PDF(Pubmed)
Abstract：
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：
在这项研究中,在西班牙接受≥2行治疗的复发/难治性弥漫性大B细胞淋巴瘤患者中,评估了改善使用axicabtageneciloleucel(axi-cel)治疗的健康影响.使用分区生存混合物治愈模型来估计每位接受axi-cel与化疗的患者的寿命累积寿命年（LYG）和质量调整寿命年（QALYs）。从用于axi-cel的ZUMA-1试验和用于化疗的SCHOLAR-1研究中提取疗效数据。在基本情况下，在接受CAR-T细胞治疗的187例患者队列中评估了axi-cel与化疗的增量结局,据“西班牙国家卫生系统高级治疗计划”报道，在基于流行病学估计的完全合格人群的替代方案中（n=490）。以目前接受axi-cel治疗的人为例，与化疗相比，axi-cel提供了额外的1341个LYG和1053个QALY。然而,当所有符合条件的患者（n=490）接受治疗时，axi-cel提供了额外的3515个LYs和2759个QALY。因此,如果所有符合条件的患者都接受了axi-cel治疗，而不是目前根据注册表进行治疗的患者（n=187），会有另外303名患者接受治疗，总共增加了2173个LYG和1706个QALY。西班牙缺乏准入导致大量LYG和QALY的损失，并应努力改善所有符合条件的患者的获取。
