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。
Abstract
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-λ between 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.
关键词
硼替佐米 /
多发性骨髓瘤 /
药品集采 /
仿制药 /
原研药 /
真实世界研究
Key words
Bortezomib /
multiple myeloma /
centralized drug procurement /
generic drug /
original drug /
real-world study
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参考文献
[1] GODFREY J, BENSON D M JR. The role of natural killer cells in immunity against multiple myeloma[J]. Leuk lymphoma, 2012, 53(9):1666-1676.
[2] MILLER KD, GODING SAUER A, ORTIZ AP, et al.Cancer statistics for Hispanics/Latinos, 2018[J]. CA Cancer J Clin, 2018, 68(6): 425-445.
[3] Ye Z, Chen J, Xuan Z, et al.Subcutaneous Bortezomib might be standard of care for patients with multiple myeloma: a systematic review and meta-analysis[J]. Drug design development and therapy, 2019, 13: 1707-1716.
[4] SAMALA RENATO V.Palliative care in patients with multiple myeloma[J]. Journal of pain and symptom management, 2019, 58(6):1113-1118.
[5] 伊佳,徐德铎,侯幸赟,等.硼替佐米治疗多发性骨髓瘤的临床综合评价[J].上海医药,2024,45(1):33-38.
[6] 罗巧,王之舟,刘文娜,等.格列美脲集采中选仿制药与原研药临床效果的真实世界研究[J].中国药业,2023,32(24):50-54.
[7] 张焕波,张岳洋,孙珮.加快完善药品和医用耗材集中带量采购制度[J].中国物价,2023(7):68-71.
[8] 李钊,陈盛,周绿林,等.国家组织药品集中带量采购的实践操作问题与优化对策[J].中国卫生经济,41(7):60-64.
[9] 邢玥,刘通,滕雪,等.曲妥珠单抗生物仿制药对比原研药治疗复发或转移性HER-2阳性乳腺癌的药物经济学评价[J].中国药房,2024,35(9):1113-1117.
[10] 郭斯伦,马韶青.美国竞争性仿制药激励制度述评及对我国的启示[J].中国药房,2023,34(13):1562-1566.
[11] HE J, HE D, HAN X, et al.Bortezomib-based regimens for newly diagnosed multiple myeloma in China: a report of 12-year real-world data[J]. Frontiers in pharmacology,2020 (12):561601.
[12] 高欣,解海谊,王慧涵,等.国产与进口硼替佐米治疗多发性骨髓瘤的真实世界疗效、安全性及经济学对比[J].现代肿瘤医学,2022,30(10):1821-1825.
[13] 邓雨琴,毛盼盼,刘璐,等.基于多中心真实世界数据的集采有效性、安全性和经济性评价——以替加环素为例[J]. 中国医疗保险,2024(5),109-115.
[14] PALUMBO A, RAJKUMAR SV,SAN MIGUEL JF,et al.International Myeloma Working Group consensus statement for the management, treatment, and supportive care of patients with myeloma not eligible for standard autologous stem-cell transplantation[J]. Journal of clinical oncology,2014,32(6):587-600.
[15] 卢洁,王宏强,陆风雁.国产硼替佐米对多发性骨髓瘤患者的疗效及对血清sFLC、LDH和ALB的影响[J].现代实用医学,2022,34(8):1036-1038.
[16] 陈园园,李英华,王东梅,等.含小剂量硼替佐米的三药联合方案治疗老年多发性骨髓瘤的临床研究[J].中国医院用药评价与分析, 2022,22(6):697-699.
[17] 国家统计局,2023年居民收入和消费支出情况[EB/OL].(2024-01-17)[2024-07-30].https://www.stats.gov.cn/sj/zxfb/202401/t20240116_1946622.html.
[18] 朱楚玉,孙维阳.常峰.我国罕见病药品价格水平和可负担性研究[J].中国药房,2021,32(8):897-903.
[19] 徐倩,徐斌.上海市口服抗帕金森病药品可负担性评价[J].中国药业,2021,30(223):1-4.
[20] 刘桂娟,公海艳,彭海英. 联合检测血清 LDH、SF、sFLC 在多发性骨髓瘤诊断中的价值[J].检验医学与临床,2023,20(16):2433-2435.
[21] 陈绿云,殷佳慧,杨岚寓,等.SUDOSCAN 检测快速有效评估硼替佐米致周围神经病变[J]. 中国肿瘤临床,2023,50(8):398-402.
