评估儿童扁桃体切除术药物干预随机临床试验的实用性与报告质量：一项系统性评价

文献信息

中文摘要

背景： 随机对照试验（RCT）是儿童扁桃体切除术围手术期和药物治疗循证实践的基础，但其真实世界实用性取决于具有临床意义的设计和透明的报告。我们应用van 't Hooft实用性框架——一个由Ioannidis所述有用临床研究特征衍生的八项标准、13个条目工具——来评价当代儿童扁桃体切除术药理学RCT的实用性和报告质量。
方法： 我们系统检索了2019年至2025年间以英文发表的、针对接受扁桃体切除术或腺样体扁桃体切除术儿童的药物干预RCT。记录由两人独立筛选（Rayyan），并由第三位评审者裁定。每项试验按两个领域（核心临床实用性和透明度）的13个条目以0-2分评分（0=未满足，1=部分满足，2=完全满足；最高综合评分26分）。问题基础条目由一名耳鼻咽喉科住院医师裁定。我们总结了条目和领域层面的满足情况，考察了时间趋势，并评估了实用性与透明度领域之间的相关性。
结果： 62项RCT符合纳入标准。综合实用性评分中位数为26分中的11分（IQR 10-15；均值11.8 ± 3.8），仅1项试验（1.6%）超过最高分的75%。大多数试验在亚洲开展（41项；66%），发表于普通医学（21项；34%）或耳鼻咽喉科（19项；31%）期刊；期刊影响因子中位数为2.00（IQR 1.30-2.90）。实用性设计几乎缺失（0项试验完全为实用性；60/62 [96.8%]为非实用性），没有试验包含性价比评估（62/62 [100%]缺失），仅7项试验（11.3%）公开了原始数据。实用性与透明度领域评分呈弱正相关（r = 0.30，p = 0.016）。综合实用性在研究期间略有增加（+0.75分/年，p = 0.018），主要由实用性领域驱动；期刊影响因子是多变量分析中唯一独立的实用性预测因素（β = 0.57，p = 0.049）。
结论： 当代儿童扁桃体切除术药理学RCT在实用性、以患者为中心的设计、经济学评价和数据透明度方面存在持续缺陷。在设计阶段系统采用van 't Hooft实用性标准和既定报告规范（SPIRIT、CONSORT）可能有助于提高这一高手术量人群未来试验的临床价值。

英文摘要

BACKGROUND: Randomized controlled trials (RCTs) underpin evidence-based perioperative and pharmacologic care in pediatric tonsillectomy, yet their real-world usefulness depends on clinically meaningful design and transparent reporting. We applied the van 't Hooft usefulness framework, an eight-criterion, 13-item instrument derived from the features of useful clinical research described by Ioannidis, to appraise the usefulness and reporting quality of contemporary pediatric tonsillectomy pharmacology RCTs.
METHODS: We systematically identified RCTs of pharmacologic interventions in children undergoing tonsillectomy or adenotonsillectomy, published in English between 2019 and 2025. Records were screened in duplicate (Rayyan) with third-reviewer adjudication. Each trial was scored on 13 items across two domains (core clinical utility and transparency) on a 0-2 scale (0 = not fulfilled, 1 = partial, 2 = full; maximum composite score 26). The problem-base item was adjudicated by an otolaryngology resident physician. We summarized item- and domain-level fulfillment, examined temporal trends, and assessed the correlation between the utility and transparency domains.
RESULTS: Sixty-two RCTs met inclusion criteria. The median composite usefulness score was 11 of 26 (IQR 10-15; mean 11.8 ± 3.8), and only 1 trial (1.6%) exceeded 75% of the maximum. Most trials were conducted in Asia (41; 66%) and published in general medicine (21; 34%) or otolaryngology (19; 31%) journals; the median journal impact factor was 2.00 (IQR 1.30-2.90). Pragmatic design was nearly absent (0 trials fully pragmatic; 60/62 [96.8%] non-pragmatic), no trial included a value-for-money assessment (62/62 [100%] absent), and only 7 trials (11.3%) made raw data available. Utility and transparency domain scores were weakly positively correlated (r = 0.30, p = 0.016). Composite usefulness increased modestly over the study period (+0.75 points/year, p = 0.018), driven by the utility domain; journal impact factor was the only independent predictor of usefulness in multivariable analysis (β = 0.57, p = 0.049).
CONCLUSION: Contemporary pediatric tonsillectomy pharmacology RCTs show consistent deficits in pragmatism, patient-centered design, economic evaluation, and data transparency. Systematic adoption of the van 't Hooft usefulness criteria and established reporting standards (SPIRIT, CONSORT) at the design stage could help improve the clinical value of future trials in this high-volume surgical population.