Department of Plastic & Reconstructive Surgery, Shanghai Ninth People's Hospital, Affiliated to Shanghai Jiao Tong University School of Medicine, 639 Zhizaoju Road, Shanghai, 200011, China.
Eur J Pediatr. 2024 Nov;183(11):5001-5011. doi: 10.1007/s00431-024-05790-2. Epub 2024 Sep 24.
A series of randomized controlled trials (RCTs) have advanced the therapeutic approaches for vascular anomalies (VA). However, a notable obstacle in applying the findings of these trials to real-world patient care is trial waste (TW). To date, the extent of TW in RCTs for VA is not clear. In June 2024, we searched the ClinicalTrials database using the entity names defined by ISSVA classification as search terms. We documented the data available and then explored PubMed and Scopus for the publication status. Reporting adequacy was evaluated using the CONSORT checklist. Design limitations were analyzed based on bias risk and whether the article referenced a relevant systematic review. One hundred fifty-nine RCTs met the inclusion criteria. The majority of RCTs focused on benign VA (81.1%) and utilized pharmacotherapy (79.9%). Over 90% of these trials were conducted in North America, Europe, and Asia as single-center studies (68.6%), with funding primarily from official institutions (83.7%). The analysis of TW excluded 61 RCTs completed after June 2020 that remained unpublished. Among the remaining 98 RCTs, 53 were published, 41 had adequate reporting, and 16 had design limitations. In total, 67 RCTs exhibited at least one characteristic of TW. The 31 RCTs without waste tended to enroll more participants (P = 0.014) and conduct studies across multiple centers (P < 0.001) and countries (P = 0.022). Multicenter participation (P = 0.028) emerged as an independent protective factor against TW.
We delineated the features of 159 VA RCTs and revealed that 68.4% of them exhibited TW. The diverse traits of the different TW indicators identified could serve as valuable insights for conducting future VA RCTs more rationally and efficiently.
• Currently, a number of RCTs have been conducted on vascular anomalies (VA). However, there has been no study analyzing the situation of trial waste in VA-related RCTs.
• This study is the first to describe the characteristics of VA-related RCTs globally over the past 20 years and has identified a high burden of trial waste in this field. Multicenter participation was an independent protective factor against trial waste.
血管异常（VA）的一系列随机对照试验（RCT）已经推进了治疗方法。然而，将这些试验的发现应用于真实患者护理的一个显著障碍是试验浪费（TW）。迄今为止，VA 的 RCT 中 TW 的程度尚不清楚。2024 年 6 月，我们使用 ISSVA 分类定义的实体名称作为搜索词，在 ClinicalTrials 数据库中进行了搜索。我们记录了可用的数据，然后在 PubMed 和 Scopus 上探索了出版物的状态。使用 CONSORT 清单评估报告的充分性。根据偏倚风险和文章是否引用了相关的系统评价，分析了设计局限性。符合纳入标准的有 159 项 RCT。大多数 RCT 侧重于良性 VA（81.1%）和药物治疗（79.9%）。这些试验中有超过 90%是在北美、欧洲和亚洲作为单一中心进行的（68.6%），主要由官方机构资助（83.7%）。TW 的分析排除了 61 项于 2020 年 6 月后完成但仍未发表的 RCT。在其余的 98 项 RCT 中，有 53 项已发表，41 项报告充分，16 项设计有局限性。总的来说，有 67 项 RCT 表现出 TW 的至少一个特征。没有浪费的 31 项 RCT 倾向于招募更多的参与者（P=0.014），并在多个中心（P<0.001）和国家（P=0.022）进行研究。多中心参与（P=0.028）是 TW 的一个独立保护因素。
我们描绘了 159 项 VA RCT 的特征，并揭示了其中 68.4%存在 TW。不同 TW 指标的不同特征可以为更合理、更有效地进行未来的 VA RCT 提供有价值的见解。
目前已经进行了多项关于血管异常（VA）的 RCT。然而，目前还没有研究分析 VA 相关 RCT 中的试验浪费情况。
这项研究首次描述了过去 20 年全球 VA 相关 RCT 的特征，并发现该领域存在大量的试验浪费。多中心参与是 TW 的一个独立保护因素。
