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

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

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。

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.