卡非佐米治疗多发性骨髓瘤的快速卫生技术评估 点击下载
| 论文标题： | 卡非佐米治疗多发性骨髓瘤的快速卫生技术评估 |
| 英文标题： | |
| 中文摘要： | 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、theCochraneLibrary、WebofScience、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。 |
| 英文摘要： | 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. |
| 期刊： | 2026年第37卷第09期 |
| 作者： | 冯振；赖冉 |
| 英文作者： | FENG Zhen，LAI Ran |
| 关键字： | 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 |
| KEYWORDS： | carfilzomib； multiple myeloma； rapid health technology assessment； effectiveness； safety； economy |
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