硼替佐米集采中选仿制药与原研药临床效果对比的真实世界研究

Real-World Study on the Clinical Efficacy Comparison Between Bid-Winning Generic and Original Drugs of Bortezomib in the Centralized Procurement

目的:基于多中心真实世界数据评估硼替佐米药品集中带量采购中选仿制药与原研药临床治疗初诊多发性骨髓瘤效果的差异。方法:回顾性收集2017年1月—2024年6月上海市5家医院电子病历系统中使用硼替佐米患者资料,筛选初治患者,按用药的不同分为集采组和原研组。使用倾向性评分匹配法对两组患者基线资料进行校正,比较两组治疗效果的直接指标及间接指标,比较两组不良事件和居民可负担情况。结果:通过1:1匹配基线资料,集采组和原研组各有215例患者。两组患者接受含硼替佐米方案治疗4疗程后的阶段性疗效相当(P>0.05),两组M蛋白、骨髓瘤浆细胞比例、β2微球蛋白、肌酐、钙和血清游离轻链sFLC-λ变化值无差异,原研组在肾功能保护上有优势(P<0.05),集采组在控制乳酸脱氢酶升高方面更优(P<0.05)。安全性方面,两组嗜睡、疼痛、周围神经病变、皮疹、过敏等发生率差异均无统计学意义(P>0.05),但原研组乏力发生率较高(P<0.05)。集采组药品居民可负担性较好。结论:硼替佐米集采中选药品的有效性和安全性总体不劣于原研药,部分指标各有优势。

Objective:The paper evaluates the difference in clinical efficacy between the bid-winning generic and original drugs in the centralized procurement of Bortezomib for the treatment of first-visit multiple myeloma based on multicenter real-world data.Methods:Data of patients treated with Bortezomib in the electronic medical record system of five hospitals in Shanghai from January 2017 to June 2024 were collected retrospectively.The first-visit patients were screened out and divided into the generic and original groups according to different medications.Patients’baseline data of the two groups were corrected by the propensity score matching method.The direct and indirect indexes of therapeutic effect,adverse reactions and residents'affordability were compared between the two groups.Results:There were 215 patients in both the generic and original groups,screened by 1:1 matching of baseline data.There was no significant difference between the two groups after 4 courses of Bortezomib-containing therapy(P>0.05).There was no significant difference in the changes of M protein,plasma cell ratio of myeloma,β2-microglobulin,creatinine,calcium and sFLC-λbetween the two groups.The original group had an advantage in protecting renal function(P<0.05),and the centralized procurement group had an advantage in controlling LDH increase(P<0.05).In terms of safety,there was no significant difference in the incidence of lethargy,pain,peripheral neuropathy,rash and allergy between the two groups(P>0.05).However,the incidence of fatigue was higher in the original group(P<0.05).The generic drugs were more affordable.Conclusion:The overall effectiveness and safety of selected drugs in the centralized procurement of Bortezomib are not inferior to the original drugs,and some indicators have their own advantages.