白蛋白结合型紫杉醇治疗转移性胰腺癌的临床综合评价  

Clinical comprehensive evaluation of albumin-bound paclitaxel in the treatment of metastatic pancreatic cancer

作　　者：赵新才 邱瑶 朱万虎 徐嵘[1] 郭澄[1] 张剑萍 Xin-Cai ZHAO;Yao QIU;Wan-Hu ZHU;Rong XU;Cheng GUO;Jian-Ping ZHANG(Department of Pharmacy,Shanghai Sixth People's Hospital Affiliated to Shanghai Jiao Tong University School of Medicine,Shanghai 200233,China)

机构地区：[1]上海交通大学医学院附属第六人民医院药剂科,上海200233

出　　处：《药物流行病学杂志》2023年第3期294-304,共11页Chinese Journal of Pharmacoepidemiology

基　　金：上海市药学会药品临床综合评价与药物治疗路径研究项目(SHYXH-ZP-2021-004)。

摘　　要：目的对白蛋白结合型紫杉醇(nab-PTX)治疗转移性胰腺癌的安全性、有效性、经济性、创新性、适宜性、可及性开展临床综合评价,为临床合理用药提供循证依据。方法通过文献综述和专家咨询法构建nab-PTX临床综合评价指标体系,并采用快速卫生技术评估(rHTA)方法对安全性、有效性和经济性进行评价,综合药品说明书、权威指南、药品价格数据以及国家药品监督管理局获得的研究资料等相关数据对创新性、适宜性和可及性进行评价。结果安全性方面,nab-PTX联合吉西他滨(GEM)方案的不良反应发生率较FOLFIRINOX方案(氟尿嘧啶+甲酰四氢叶酸+伊立替康+奥沙利铂)大多较低,但贫血和神经毒性发生率较高,与GEM方案的不良反应发生率相似,中性粒细胞减少、血小板减少的不良反应发生率高于替吉奥(S1)方案。有效性方面,nab-PTX联合GEM方案与GEM方案、S1方案比较,能显著增加客观缓解率(ORR)、疾病控制率(DCR);与FOLFIRINOX方案相比,在总生存期、无进展生存期、ORR、DCR和1年生存率指标方面未见明显差异,但显著降低了需要二线方案治疗率。经济性方面,nab-PTX联合GEM方案对比FOLFIRINOX方案治疗转移性胰腺癌更具有经济性;相比于GEM方案,nab-PTX联合GEM方案不具备成本-效果优势,但随着政策发展及nab-PTX价格降低,nab-PTX联合GEM方案经济优势将会进一步体现。在创新性方面,nab-PTX是由白蛋白结合紫杉醇形成纳米颗粒的全新结构,使得其更具优势。在适宜性方面,患者使用依从性提高,临床使用过程也具有较好的适宜性。在可及性方面,nabPTX的价格水平稳定,可获得性较好,且近年来价格降价,可负担性较好。结论nabPTX属于紫杉醇制剂的创新结构,nab-PTX联合GEM方案治疗转移性胰腺癌患者的安全性和有效性较好,不劣于经典FOLFIRINOX方案,并且比FOLFIRINOX方案更具有经济性,患者使用依从性提高,临床�Objective To carry out the clinical comprehensive evaluation of the safety,effectiveness,economy,innovation,suitability and accessibility of albumin-bound paclitaxel(nab-PTX)in the treatment of metastatic pancreatic cancer and to provide evidencebased reference for drug rational use.Methods The comprehensive evaluation index system of nab-PTX was constructed by means of literature reviewed and expert consultation.Based on the constructed clinical comprehensive evaluation index system,the method of rapid health technology assessment(rHTA)was used to evaluate the safety,effectiveness and economy.In terms of innovation,suitability and accessibility evaluation,relevant data such as drug package inserts,authoritative guidelines,drug price data and research data obtained by the National Medical Products Administration were evaluated.Results The results of all dimensions showed that in terms of safety,nab-PTX combined gemcitabine(GEM)regimen had a lower incidence of adverse reactions than that of FOLFIRINOX regimen(fluorouracil+leucovorin+irinotecan+oxaliplatin),similar to that of GEM regimen,and had a higher incidence of neutropenia and thrombocytopenia than S1.In terms of effectiveness,nab-PTX combined GEM regimen could significantly increase objective response rate(ORR)and disease control rate(DCR)compared with GEM and S1 regimen.Compared with FOLFIRINOX regimen,nab-PTX combined GEM regimen showed no significant difference in overall survival,progression-free survival,ORR,DCR and 1-year survival,but significantly reduced the rate of requiring second-line treatment.In terms of economy,nab-PTX combined GEM regimen was more economical than FOLFIRINOX regimen in the treatment of metastatic pancreatic cancer.What’s more,the nabPTX combined GEM regimen has no cost-effectiveness advantage,but with the development of policies and the reduction of the nab-PTX price,the economic advantage of nab-PTX combined GEM regimen will be further reflected.In terms of innovation,nab-PTX was a new structure of nanoparticles formed by co

关 键 词：白蛋白结合型紫杉醇 转移性胰腺癌 临床综合评价 快速卫生技术评估 

分 类 号：R735.9[医药卫生—肿瘤]