药物治疗老年急性髓系白血病临床研究评价指标分析

Analysis of clinical evaluation outcomes of drug treatment for elderly acute myeloid leukemia

关键词:  老年急性髓系白血病  有效性指标  安全性指标  核心指标集  随机对照试验

Key words:  elderly acute myeloid leukemia  effectiveness outcomes  safety outcomes  core outcome set  randomized controlled trial

摘要:

[目的] 分析药物治疗老年急性髓系白血病（AML）临床随机对照试验（RCT）的评价指标，为构建老年AML临床试验的核心指标集奠定基础。[方法] 计算机检索中国期刊全文数据库（CNKI）、万方数据库（WanFang Data）、中国生物医学文献数据库（SinoMed）、PubMed、The Cochrane Library、EMbase数据库以及中国临床试验注册中心、美国临床试验数据库（ClinicalTrials.gov）平台中药物治疗老年AML的RCTs，检索时限为2015年1月1日—2020年11月1日。对评价指标进行标化及归类，并进行描述性统计。[结果] 共纳入64个RCTs，干预措施治疗1~4个疗程，总体治疗时间7~63 d不等，最长随访时间5年，最短随访1个月。共报告168个评价指标，合计使用252次，经规范化为38个评价指标，按功能属性归为6个指标域。各个研究采用的评价指标疗效判定标准不一致，指标的测量时点报告也较少。[结论] 目前老年AML临床研究的评价指标，存在指标选择不合理；名称使用不规范；测量时点报告不清、选择不合理等问题。亟需建立符合疾病特质和干预措施特殊性的药物治疗老年AML核心指标集，为相关临床试验的设计和实施提供参考和依据。

Abstract:

[Objective] To analyze the efficasy evaluation outcomes of randomized controlled trials (RCTs) on drug therapy for elderly acute myeloid leukemia (AML), and to establish the basis for core outcome set (COS)about elderly AML.[Methods] CNKI, WanFang, SinoMed, PubMed, Cochrane library, EMbase databases, ChiCTR and ClinicalTrials.gov were searched by computer, and the clinical randomized controlled trial(RCT) for the drug treatment of elderly AML treatment comprehensively collected. The retrieval time was set on January 1, 2015 to November 1, 2020. Literature screening and data extraction were conducted independently by 2 system evaluators according to inclusion and exclusion criteria.[Results] A total of 64 RCTs were included, and the intervention course was observed for 1~4 treatment courses, with treatment duration ranging from 7~63 days. The longest follow-up was 5 years, and the shortest follow-up was 1 month. A total of 168 evaluation outcomes were reported, and 252 of them were used in total, which were normalized to 38 evaluation outcomes and classified into 6 outcomes domains according to functional attributes. In addition, the evaluation outcomes used in this study have inconsistent criteria for the efficacy evaluation, and there are few reports on the measurement of the outcomes.[Conclusion] The evaluation outcomes of clinical research on elderly AML are not reasonable now. Definition of outcomes are not standard. The measurement report is not clear, the choices of outcomes is not reasonable and so on. There is an urgent need to establish core outcome set (COS)about elderly AML durg treating with the characteristics of the disease and the specificity of the intervention, so as to provide reference and basis for the design and implementation of clinical trials.