CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CAR-T(CAR-T)细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:The Health Impacts of Better Access to Axicabtagene Ciloleucel: The Case of Spain.
The Health Impacts of Better Access to Axicabtagene Ciloleucel: The Case of Spain.
分数与星级只用于站内排序 —— 不代表疗效、安全性或个人适用性。
本研究评估了在西班牙接受过2线治疗的复发/难治性弥漫性大B细胞淋巴瘤患者中,改善axicabtagene ciloleucel(axi-cel)治疗可及性所带来的健康影响。采用分区生存混合治愈模型,估算接受axi-cel治疗与化疗相比,每例患者终生累积获得的生命年(LYG)和质量调整生命年(QALYs)。axi-cel的疗效数据来自ZUMA-1试验,化疗的疗效数据来自SCHOLAR-1研究。在基础情景中,根据“西班牙国家卫生系统先进疗法计划”报告,在187例接受CAR-T 细胞治疗的患者队列中评估了axi-cel相对于化疗的增量结局;在替代情景中,则基于流行病学估计,在全部符合条件人群中(n = 490)进行评估。以目前接受axi-cel治疗的患者来看,与化疗相比,axi-cel额外提供了1341个LYGs和1053个QALYs。
然而,如果所有符合条件患者(n = 490)均接受治疗,axi-cel将额外提供3515个LYs和2759个QALYs。
因此,如果所有符合条件患者均接受axi-cel治疗,而不是按登记处目前接受治疗的患者(n = 187),则将额外有303例患者接受治疗,总计额外增加2173个LYGs和1706个QALYs。西班牙缺乏可及性已导致大量LYGs和QALYs损失,应努力改善所有符合条件患者的可及性。
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.
在 PubMed 查看 → 出版商原文(DOI) 全文 PDF(PMC)· 可下载 治疗专题与资料阅读指南 资料来源与翻译说明 报告译文或资料问题 →
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