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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
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