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卡非佐米治疗多发性骨髓瘤的快速卫生技术评估
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冯 振 ,赖 冉(徐州医科大学附属医院药学部,江苏 徐州 221006)
中图分类号 R979.1;R551.3 文献标志码 A 文章编号 1001-0408(2026)09-1222-07
DOI 10.6039/j.issn.1001-0408.2026.09.20
摘 要 目的 综合分析卡非佐米治疗多发性骨髓瘤(MM)的有效性、安全性和经济性,为其临床合理用药提供循证参考。方法
系统检索PubMed、Embase、the Cochrane Library、Web of Science、中国知网、万方数据库、维普网等数据库,同时涵盖卫生技术评估
相关网站,检索时间为建库至2024年12月31日。两位研究者依据预设的纳入与排除标准独立筛选文献,提取数据并评价文献质
量,最后对数据进行描述性统计分析。结果 共纳入21篇文献,包括系统评价/Meta分析7篇、临床试验5篇、药物经济学研究5篇
以及卫生技术评估报告4份。有效性分析显示,与对照措施相比,卡非佐米方案可以显著提高总缓解率和临床获益率(P<0.05),
延长无进展生存期并改善患者生活质量(P<0.05),但对总生存期的影响尚需进一步证实。安全性分析显示,与对照措施相比,卡
非佐米方案在周围神经病变风险方面的差异无统计学意义,但心脏毒性和高血压的发生风险显著增加(P<0.05)。经济性分析显
示,卡非佐米方案在美国和欧洲部分国家的二线治疗中具有一定的成本-效益优势,但其经济性受药品价格、给药方案及地区差异
影响,在部分国家或三线治疗中可能缺乏优势。结论 卡非佐米在MM治疗中有效性明确、安全性总体可接受,但需警惕心脏毒性
和高血压风险。卡非佐米的治疗价格较高,亟需开展中国人群的药物经济学研究。
关键词 卡非佐米;多发性骨髓瘤;快速卫生技术评估;有效性;安全性;经济性
Rapid health technology assessment of carfilzomib in the treatment of multiple myeloma
FENG Zhen,LAI Ran(Dept. of Pharmacy, the Affiliated Hospital of Xuzhou Medical University, Jiangsu
Xuzhou 221006, China)
ABSTRACT OBJECTIVE To comprehensively analyze the effectiveness, safety and economic value of carfilzomib in the
treatment of multiple myeloma (MM), and provide evidence-based guidance for clinical rational drug use. METHODS A
systematic search was performed in PubMed, Embase, the Cochrane Library, Web of Science, CNKI, Wanfang, VIP, and
relevant health technology assessment (HTA) websites, from database inception to December 31, 2024. Two researchers
independently screened studies based on predefined inclusion and exclusion criteria, extracted data, and assessed study quality.
Descriptive statistical analyses were conducted. RESULTS A total of 21 studies were included, comprising 7 systematic reviews/
meta-analyses, 5 clinical trials, 5 pharmacoeconomic studies, and 4 HTA reports. Effectiveness analysis indicated that, compared
with control groups, carfilzomib significantly improved overall response rate and clinical benefit rate (P<0.05), prolonged
progression-free survival, and enhanced patient’s quality of life (P<0.05), while its impact on overall survival remains to be
further confirmed. Safety analysis showed there was no significant difference in the risk of peripheral neuropathy between
carfilzomib and controls, while the risks of cardiotoxicity and hypertension were significantly increased (P<0.05). Economic
analysis suggested that carfilzomib regimens demonstrated certain cost-effectiveness in second-line treatment in the U.S. and some
European countries. However, its economic value was influenced by drug price, dosing regimen, and regional differences, and it
may lack a cost advantage in some countries or in third-line treatment. CONCLUSIONS Carfilzomib shows definite efficacy and
generally acceptable safety in the treatment of MM, but cardiotoxicity and hypertension risks require careful monitoring. Given its
high treatment cost, pharmacoeconomic studies in the Chinese population are urgently needed.
KEYWORDS carfilzomib; multiple myeloma; rapid health technology assessment; effectiveness; safety; economy
Δ 基金项目 江苏省药品临床综合评价项目(No.WJ20221502);江
多发性骨髓瘤(multiple myeloma,MM)是常见的血
苏 省 新 药 研 究 与 临 床 药 学 重 点 实 验 室 2025 年 度 开 放 课 题(No.
25KF24) 液系统恶性肿瘤,以骨髓内单克隆浆细胞异常增生为特
*第一作者 主管药师,硕士。研究方向:临床药学、药物经济学。 [1]
征,可累及多器官系统 。在血液系统肿瘤中,MM发病
电话:0516-85806331。E-mail:735502926@qq.com
[2]
率居第2位,其在我国的发病率约1.6/10万 。MM可导
# 通信作者 副主任药师,硕士。研究方向:临床药学。电话:
0516-85806331。E-mail:xz598679882@163.com 致骨破坏、肾功能损害、贫血及免疫功能异常等严重并
· 1222 · China Pharmacy 2026 Vol. 37 No. 9 中国药房 2026年第37卷第9期

