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硼替佐米治疗多发性骨髓瘤的成本效益:一项系统评价

Cost-effectiveness of bortezomib for multiple myeloma: a systematic review.

作者信息

Chen Wendong, Yang Yicheng, Chen Yi, Du Fen, Zhan Huan

机构信息

Normin Health, Toronto, ON, Canada.

Xian Janssen, Beijing, People's Republic of China.

出版信息

Clinicoecon Outcomes Res. 2016 May 3;8:137-51. doi: 10.2147/CEOR.S104195. eCollection 2016.

Abstract

OBJECTIVES

To review published cost-effectiveness analyses (CEA) assessing bortezomib (BTZ) for multiple myeloma (MM) and explore possible bias affecting the cost-effectiveness of BTZ.

METHODS

Literature was searched for published CEAs assessing BTZ or BTZ-containing regimens for MM from 2003 to 2015. The reported incremental cost-effectiveness ratios (ICER) were adjusted by 2014 country-specific gross domestic product per capita (GDPPC) to compare the cost-effectiveness threshold of the World Health Organization (3 GDPPC per gained quality-adjusted life year [QALY]).

RESULTS

A total of 17 published CEAs were included in this review. When compared to non-BTZ treatments, BTZ-containing regimens were cost-effective for induction treatment prior to stem cell transplantation (SCT) in Canada, Poland, and Germany (ICER per QALY: 0.9299-2.254 GDPPC). BTZ/melphalan/prednisolone (VMP) was cost-effective for previously untreated and SCT-ineligible MM patients when compared to melphalan plus prednisolone (MP), melphalan/prednisone/lenalidomide with lenalidomide maintenance, and cyclophosphamide/thalidomide/dexamethasone (CTD) (ICER per QALY: dominant to 2.374 GDPPC) in Canada, UK, and USA. BTZ was cost-effective for relapsed/refractory MM when compared to best supportive care (ICER per life year: 0.9317-1.8210 GDPPC) in the UK and the USA, thalidomide in USA (0.5178 GDPPC/LY), and dexamethasone (DEX) in four Nordic countries (€54,451-€81,560/QALY). However, the cost-effectiveness for VMP versus MP plus thalidomide (MPT) and continuous lenalidomide (LEN) plus low-dose DEX (RD) for previously untreated and SCT-ineligible MM patients and BTZ versus LEN/DEX for relapsed/refractory MM patients could be unreliable because of the bias associated with model design and the indirect comparisons of treatment effects.

CONCLUSION

Published CEAs suggested that BTZ or BTZ-containing regimens were cost-effective when compared to most non-BTZ treatments for MM. However, the conflicting cost-effectiveness for VMP versus MPT for previously untreated and SCT-ineligible MM and BTZ versus LEN/DEX for relapsed/refractory MM needs more robust evidence for further clarification.

摘要

目的

回顾已发表的评估硼替佐米(BTZ)用于治疗多发性骨髓瘤(MM)的成本效益分析(CEA),并探讨可能影响BTZ成本效益的偏差。

方法

检索2003年至2015年期间已发表的评估BTZ或含BTZ方案治疗MM的CEA文献。根据2014年各国人均国内生产总值(GDPPC)对报告的增量成本效益比(ICER)进行调整,以比较世界卫生组织的成本效益阈值(每获得一个质量调整生命年[QALY]3倍GDPPC)。

结果

本综述共纳入17篇已发表的CEA。与非BTZ治疗相比,在加拿大、波兰和德国,含BTZ方案用于干细胞移植(SCT)前的诱导治疗具有成本效益(每QALY的ICER:0.9299 - 2.254倍GDPPC)。与美法仑加泼尼松(MP)、美法仑/泼尼松/来那度胺联合来那度胺维持治疗以及环磷酰胺/沙利度胺/地塞米松(CTD)相比,硼替佐米/美法仑/泼尼松(VMP)方案对于既往未治疗且不符合SCT条件的MM患者具有成本效益(每QALY的ICER:从占优到2.374倍GDPPC),在加拿大、英国和美国均如此。与最佳支持治疗相比,BTZ用于复发/难治性MM具有成本效益(每生命年的ICER:0.9317 - 1.8210倍GDPPC),在英国和美国,与沙利度胺相比(0.5178倍GDPPC/LY),以及在四个北欧国家与地塞米松(DEX)相比(每QALY 54,451 - 81,560欧元)。然而,对于既往未治疗且不符合SCT条件的MM患者,VMP与MP加沙利度胺(MPT)以及持续来那度胺(LEN)加低剂量DEX(RD)相比的成本效益,以及对于复发/难治性MM患者,BTZ与LEN/DEX相比的成本效益可能不可靠,因为存在与模型设计相关的偏差以及治疗效果的间接比较。

结论

已发表的CEA表明,与大多数治疗MM的非BTZ方案相比,BTZ或含BTZ方案具有成本效益。然而,对于既往未治疗且不符合SCT条件的MM患者,VMP与MPT相比以及复发/难治性MM患者BTZ与LEN/DEX相比存在相互矛盾的成本效益,需要更有力的证据来进一步阐明。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d601/4861606/2d652245ba1c/ceor-8-137Fig1.jpg

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