尼洛替尼治疗慢性髓细胞性白血病的快速卫生技术评估

Nilotinib for the treatment of chronic myelogenous leukemia:a rapid health technology assessment

目的:基于快速卫生技术评估(health technology assessment,HTA)方法,评价尼洛替尼治疗伊马替尼耐药/不耐受的慢性髓细胞性白血病(chronic myelogenous leukemia,CML)的有效性、安全性和经济性,为临床药物选择和决策者提供循证依据。方法:通过计算机检索Pubmed,Embase和the Cochrane Library英文数据库和中国知网(CNKI)、万方中文数据库,由2名评价员根据纳入、排除标准独立检索、筛选和提取数据结果,并对结果进行定性分析。结果:共纳入1篇HTA报告,7篇药物经济学研究。使用尼洛替尼治疗伊马替尼耐药/不耐受的CML患者可以更快地产生细胞应答,使患者从中获益,减少疾病进展,并能获得更长的生存期。其不良反应主要为中性粒细胞减少、血小板减少和皮疹等。尼洛替尼与达沙替尼及大剂量伊马替尼相比可以延长患者质量调整生命年(quality-adjusted life year,QALY)并具有较好的成本效果。结论:尼洛替尼治疗伊马替尼耐药/不耐受的CML患者具有良好的有效性、安全性和经济性。

Objective:To analyze the effectiveness,safety and economy of nilotinib in the treatment of patients with imatinib-resistant or-intolerant chronic myelogenous leukemia(CML),so as to provide currently available best evidence for decision makers of health policies in medication selection.Methods:The study included a systematic literature search for the studies published in English databases(PubMed,Embase,Cochrane Library)and Chinese databases(CNKI and Wan Fang databases).The results were independently retrieved,screened and extracted by two reviewers based on inclusion and exclusion criteria.Results:A health technology assessment(HTA)report and seven cost-effective studies were finally included.Faster and deeper molecular responses were observed in the patients with imatinib-resistant or-intolerant CML when using nilotinib and disease progression reduced,making more patients benefit from it.Besides,the main adverse reactions of the drug were neutropenia,thrombocytopenia and rash.Nilotinib can prolong the quality-adjusted life year(QALY)with better cost-effectiveness compared with dasatinib and high-dose imatinib.Conclusion:Nilotinib is effective,safe and economical in the treatment of patients with imatinib-resistant/-intolerant CML.