Chen Wendong, Yang Yicheng, Chen Yi, Du Fen, Zhan Huan
Normin Health, Toronto, ON, Canada.
Xian Janssen, Beijing, People's Republic of China.
Clinicoecon Outcomes Res. 2016 May 3;8:137-51. doi: 10.2147/CEOR.S104195. eCollection 2016.
To review published cost-effectiveness analyses (CEA) assessing bortezomib (BTZ) for multiple myeloma (MM) and explore possible bias affecting the cost-effectiveness of BTZ.
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]).
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.
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.
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。
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)。
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。
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。