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[摘要]
目的 系统评估儿童功能性腹痛药物临床试验的设计要素,通过标准化试验设计,为不同试验结果的比较提供一定的可行性。方法 检索PubMed、Cochrane以及EMBASE数据库,纳入儿童功能性腹痛的随机对照临床研究文献,采用罗马诊断标准,干预措施为药物,语种为英文。结果 检索出文献561篇,最终纳入14项研究,均以改善腹痛及其相关症状为主要试验目的,其中改善腹痛12项(85.71%);均采用随机、安慰剂对照、优效性检验,13项为双盲,1项未提及,8项设计了样本量估算,5项为多中心试验;诊断采用罗马Ⅱ标准4项(28.57%),罗马Ⅲ标准10项(71.43%);干预措施依次为益生菌9项(64.29%)、抗抑郁药2项(14.29%),以及抗过敏药、解痉药、促胃肠动力药各1项(各占7.14%);6项设计了导入期,时长为1~4周;疗程为2周1项(7.14%),4周10项(71.43%),8周2项(14.29%),12周1项(7.14%);10项设计了随访,时长为4周~6个月;5项(35.71%)的主要有效性指标为腹痛程度和据此定义的治疗成功或应答,4项(28.57%)为腹痛程度、频率,3项(21.43%)为腹痛程度、频率和持续时间,2项为基于量表的症状改善情况;腹痛评价,采用面部表情量表法(WBS或FPS)9项(64.29%),视觉模拟评分量表(VAS)或疼痛数字评分法(NRS-11)5项(35.71%),同时采用VAS和FPS1项(7.14%)。全部研究的主要安全性指标均为不良事件的发生率。结论 纳入研究的文献信息完善、质量较高,结果涵盖了儿童功能性腹痛临床研究设计的基本要素,具有一定的借鉴价值。
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[Abstract]
Objective To systematically evaluate the design points of clinical trials of children with functional abdominal pain, and to provide certain feasibility for comparison with different trail results through standardized trail design. Methods The PubMed, Cochrane and EMBASE databases were searched. Studies were included if it was a randomized controlled clinical study of children with functional abdominal pain. The Rome diagnostic criteria were used, the intervention was a drug and the language was English. Results A total of 561 articles were found and 14 were eventually included. All 14 studies were aimed at improving abdominal pain and related symptoms, including 12 (85.71%) cases of abdominal pain. All studies were randomized, placebo-controlled, and superiority trail. 13 items were double-blind, one item was not mentioned, eight items were designed for sample size estimation, and five items were multi-central trial; Roman Ⅱ standard was used for four (28.57%) items, Rome Ⅲ standard in 10 (71.43%) items; interventions followed by probiotics 9 (64.29%), antidepressants 2 (14.29%), and one item with antiallergic, antispasmodic and gastrointestinal motility drugs (7.14% each); six studies designed the introduction period, the duration was 1-4 weeks; the course of treatment was one (7.14%) item at 2 weeks, 10 (71.43%) item at 4 weeks, 2 (14.29%) items at 8 weeks and one (7.14%) item at 12 weeks; 10 studies designed follow-up with duration 4 weeks to 6 months; the main efficacy indicators of 5 (35.71%) items were the degree of abdominal pain and the treatment success or response based on it, 4 (28.57%) items were the degree and frequency of abdominal pain, 3 (21.43%) items were the degree, frequency and duration of abdominal pain, and 2 items were based on the improvement symptoms of the scale. In abdominal pain evaluation, 9 (64.29%) items were usd facial expression scale method (WBS or FPS), 5 (35.71%) items by visual analog scale score (VAS) or pain digital score method (NRS-11), and one (7.14%) item by using VAS and FPS 1. The main safety indicators for all studies were the incidence rate of adverse events. Conclusion The literature information included in the study is perfect and the quality is high. The results cover the basic elements of clinical research design of functional abdominal pain in children, and have certain reference value.
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