Khanpour Ardestani Samaneh, Karkhaneh Mohammad, Yu Hai Chuan, Hydrie Muhammad Zafar Iqbal, Vohra Sunita
CARE Program, Department of Pediatrics, Faculty of Medicine & Dentistry, University of Alberta, Edmonton, Alberta, Canada.
Directorate of Public Health, Ministry of Health Jeddah Region, Jeddah, Saudi Arabia.
BMJ Open. 2017 Dec 19;7(12):e014610. doi: 10.1136/bmjopen-2016-014610.
Our objective was to systematically review randomised clinical trials (RCTs) of paediatric type 1 diabetes mellitus (T1DM) to assess reporting of (1) primary outcome, (2) outcome measurement properties and (3) presence or absence of adverse events.
Electronic searches in MEDLINE, EMBASE, CINAHL, Cochrane SR and the Cochrane Central Register of Controlled Trials (CENTRAL) databases were undertaken. The search period was between 2001 and 2017. English-language RCTs on children younger than 21 years with T1DM were selected. We excluded studies of diagnostic or screening tools, multiple phase studies, protocols, and follow-up or secondary analysis of data.
Of 11 816 unique references, 231 T1DM RCTs were included. Of total 231 included studies, 117 (50.6%) trials failed to report what their primary outcome was. Of 114 (49.4%) studies that reported primary outcome, 88 (77.2%) reported one and 26 (22.8%) more than one primary outcomes. Of 114 studies that clearly stated their primary outcome, 101 (88.6%) used biological/physiological measurements and 13 (11.4%) used instruments (eg, questionnaires, scales, etc) to measure their primary outcome; of these, 12 (92.3%) provided measurement properties or related citation. Of the 231 included studies, 105 (45.5%) reported that adverse events occurred, 39 (16.9%) reported that no adverse events were identified and 87 (37.7%) did not report on the presence or absence of adverse events.
Despite tremendous efforts to improve reporting of clinical trials, clear reporting of primary outcomes of RCTs for paediatric T1DM is still lacking. Adverse events due to DM interventions were often not reported in the included trials. Transparent reporting of primary outcome, validity of measurement tools and adverse events need to be improved in paediatric T1DM trials.
我们的目的是系统评价1型糖尿病（T1DM）儿科患者的随机临床试验（RCT），以评估（1）主要结局、（2）结局测量特性以及（3）不良事件的有无情况的报告。
对MEDLINE、EMBASE、CINAHL、Cochrane系统评价（SR）以及Cochrane对照试验中央注册库（CENTRAL）数据库进行电子检索。检索时间段为2001年至2017年。选取关于21岁以下T1DM儿童的英文RCT。我们排除了诊断或筛查工具研究、多阶段研究、方案以及数据的随访或二次分析。
在11816条独特参考文献中，纳入了231项T1DM RCT。在总共231项纳入研究中，117项（50.6%）试验未报告其主要结局是什么。在报告了主要结局的114项（49.4%）研究中，88项（77.2%）报告了1个主要结局，26项（22.8%）报告了不止1个主要结局。在114项明确陈述了其主要结局的研究中，101项（88.6%）使用生物学/生理学测量方法，13项（11.4%）使用工具（如问卷、量表等）来测量其主要结局；其中，12项（92.3%）提供了测量特性或相关引文。在231项纳入研究中，105项（45.5%）报告发生了不良事件，39项（16.9%）报告未发现不良事件，87项（37.7%）未报告不良事件的有无情况。
尽管为改善临床试验报告付出了巨大努力，但儿科T1DM的RCT主要结局的清晰报告仍然缺乏。纳入试验中常常未报告糖尿病干预导致的不良事件。儿科T1DM试验需要改善主要结局的透明报告、测量工具的有效性以及不良事件的报告情况。
