• 中国核心期刊(遴选)数据库收录期刊
  • 中文科技期刊数据库收录期刊
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  • 中国学术期刊综合评价数据库统计源期刊等

中国药物评价 ›› 2021, Vol. 38 ›› Issue (3): 183-190.

• 专栏——儿童药临床综合议价 • 上一篇    下一篇

达沙替尼治疗儿童费城染色体阳性急性淋巴细胞白血病的临床综合评价

曹旺1ab, 尉耘翠1ab, 刘璐2, 王晓玲1a, 张瑞东1c, 史黎炜3, 邱英鹏3, 肖月3, 贾露露1ab*   

  1. 1a. 国家儿童医学中心 首都医科大学附属北京儿童医院药学部, 北京 100045;
    1b. 国家儿童医学中心 首都医科大学附属北京儿童医院临床研究中心, 北京 100045;
    1c. 国家儿童医学中心 首都医科大学附属北京儿童医院血液一科, 北京 100045;
    2. 青岛市妇女儿童医院药学部, 山东 青岛 266011;
    3. 国家卫生健康委卫生发展研究中心, 北京 100044
  • 收稿日期:2021-07-20 修回日期:2021-04-29 出版日期:2021-06-28 发布日期:2021-06-28
  • 基金资助:
    国家卫生健康委药政司药品临床综合评价指南开发课题;国家重大新药创制专项“儿童示范性新药临床评价技术平台建设”(2017ZX09304029)

Comprehensive Clinical Evaluation of Dasatinib in the Treatment of Children with Philadelphia-positive Acute Lymphoblastic Leukemia

  1. 1a. Department of Pharmacy, Beijing Children's Hospital, Capital Medical University, National Center
         for Children's Health, Beijing 100045, China;
    1b. Department of Pharmacy, Beijing Children's Hospital, Capital Medical University, National Center
         for Children′s Health, Beijing 100045, China;
    1c. Department of Hematology, Beijing Children's Hospital, Capital Medical University, National Center
         for Children′s Health, Beijing 100045, China;
    2. Department of Pharmacy, Qingdao Women and Children′s Hospital, Qingdao, Shandong 266011, China;
    3. National Health Development Research Center, National Health Commission, Beijing 100044, China
  • Received:2021-07-20 Revised:2021-04-29 Online:2021-06-28 Published:2021-06-28

摘要: 目的:评价达沙替尼治疗儿童费城染色体阳性急性淋巴细胞白血病的临床价值,为药品说明书修订、临床合理选药提供依据。方法: 采用文献研究法、回顾性队列研究法、药物经济学模型研究法,比较达沙替尼与伊马替尼治疗儿童费城染色体阳性急性淋巴细胞白血病的有效性、安全性、经济性。结果: 达沙替尼治疗的有效性优于伊马替尼,安全性无显著差异,且具有潜在的经济学优势。结论: 达沙替尼在有效性、经济性上均优于伊马替尼,且安全性无明显差异,因此推荐其用于儿童费城染色体阳性急性淋巴细胞白血病(Ph+ALL)的临床治疗。本次评价尽管存在局限性,但通过试点验证了《儿童药品临床综合评价技术指南》的适用性和科学性,特别是如何基于我国临床实际用药数据进行药品临床价值分析。

关键词: font-size:13.3333px, ">达沙替尼;儿童费城染色体阳性急性淋巴细胞白血病;药品临床综合评价;方法学

Abstract: Objective: To evaluate the clinical value of dasatinib in the treatment of children with Philadelphia-positive acute lymphoblastic leukemia, and to provide basis for drug label revision and rational drug selection in clinic. Methods: Literature review, retrospective cohort study and pharmacoeconomic model were used to compare the efficacy, safety and economy of dasatinib and imatinib in the treatment of children with Philadelphia-positive acute lymphoblastic leukemia. Results: The efficacy of dasatinib treatment was superior to imatinib. The dasatinib had also the potential economic advantages and there was no significant difference in safety. Conclusion: Dasatinib is superior to imatinib in efficacy and economy, and there is no significant difference in safety. Therefore, it is recommended for the clinical treatment of children with pH + ALL. Although multi-dimensional evidences were collected in this evaluation, the quantity and quality of evidences were not enough, and the conclusions needed to be verified in a larger scope. In the next step, multi-center real world studies could be carried out to rigorously demonstrate the clinical value of relevant drugs and provide evidence for drug policy formulation.
  

Key words: font-size:13.3333px, ">Dasatinib; Philadelphia-positive acute lymphoblastic leukemia in children; Drug clinical comprehensive evaluation; Methodology

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