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儿童 ALL 免疫治疗的成本和成本效果:一项系统综述

英文原题:Cost and cost-effectiveness of immunotherapy in childhood ALL: A systematic review.

查看英文原题

Cost and cost-effectiveness of immunotherapy in childhood ALL: A systematic review.

PubMed 2023/11/13(内容时间) EJHaem Q4 · IF 1.3(JCR 2025)

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中文摘要

儿童急性淋巴细胞白血病(pALL)的生存率已显著提高;复发/难治性(r/r)急性淋巴细胞白血病(ALL)仍然具有挑战性。免疫疗法是r/r ALL快速发展的治疗方法,但成本效果数据有限。

本研究识别了pALL中免疫疗法的现有经济学评价,并总结了成本效果。检索了Medline、Embase和其他数据库,从建库至2022年10月。纳入评估pALL中免疫疗法的成本效果分析。成本以2021年美元报告。在2960项研究中,11项符合纳入标准。Tisagenlecleucel与标准治疗、clofarabine单药治疗、clofarabine联合治疗或blinatumomab进行了比较。尚无研究评估blinatumomab或inotuzumab ozogamicin。六项研究发现tisagenlecleucel具有成本效果,其中五项由Novartis支持。四项发现其具有成本效果的潜力,一项发现其不具有成本效果。Tisagenlecleucel的成本效果高度依赖于标价和治愈率。

本研究可为tisagenlecleucel在pALL中的使用提供信息。

展开英文摘要原文

Survival rates for pediatric acute lymphoblastic leukemia (pALL) have improved dramatically; relapsed/refractory (r/r) acute lymphoblastic leukemia (ALL) remains challenging. Immunotherapies are rapidly evolving treatments for r/r ALL with limited cost-effectiveness data.

This study identifies existing economic evaluations of immunotherapy in pALL and summarizes cost-effectiveness. Medline, Embase, and other databases were searched from inception to October 2022. Cost-effectiveness analyses evaluating immunotherapy in pALL were included. Costs reported in 2021 USD. Of 2960 studies, 11 met inclusion criteria. Tisagenlecleucel was compared to standard of care, clofarabine monotherapy, clofarabine combination therapy, or blinatumomab.

No studies have evaluated blinatumomab or inotuzumab ozogamicin. Six studies found tisagenlecleucel to be cost-effective, five of which were supported by Novartis. Four found that it had the potential to be cost-effective, and one found that it was not cost-effective. The cost-effectiveness of tisagenlecleucel was highly dependent on list price and cure rates.

This study can inform the use of tisagenlecleucel in pALL.

论文信息

作者
Scoleri-Longo Y、Pechlivanoglou P、Gupta S
第一作者单位
Department of Paediatrics Post Graduate Medical Education The Hospital for Sick Children Toronto Ontario Canada.Canada
通讯作者单位
Cancer Research Program Institute for Clinical Evaluative Sciences Toronto Ontario Canada.Canada
文献类型
综述
期刊
EJHaem2024 Feb
原文标识
PubMed 38406535 · DOI 10.1002/jha2.814