Desai Bijal, Desai Veeral, Shah Shivani, Srinath Archita, Saleh Amr, Simunovic Nicole, Duong Andrew, Sprague Sheila, Bhandari Mohit
Faculty of Health Sciences, McMaster University, Hamilton, ON, Canada.
Schulich School of Medicine and Dentistry, Western University, London, ON, Canada.
BMC Musculoskelet Disord. 2018 Nov 24;19(1):412. doi: 10.1186/s12891-018-2337-7.
The primary objective of this systematic review is to examine the characteristics of pilot randomized controlled trials (RCTs) in the orthopaedic surgery literature, including the proportion framed as feasibility trials and those that lead to definitive RCTs. This review aim to answer the question of whether pilot RCTs lead to definitive RCTs, whilst investigating the quality, feasibility and overall publication trends of orthopaedic pilot trials.
Pilot RCTs in the orthopaedic literature were identified from three electronic databases (EMBASE, MEDLINE, and Pubmed) searched from database inception to January 2018. Search criteria included the evaluation of at least one orthopaedic surgical intervention, research on humans, and publication in English. Two reviewers independently screened the pool of pilot trials, and conducted a search for corresponding definitive trials. Screened pilot RCTs were assessed for feasibility outcomes related to efficiency, cost, and/or timeliness of a large-scale clinical trial involving a surgical intervention. The quality of the pilot and definitive trials were assessed using the Checklist to Evaluate a Report of a Non-Pharmacological Trial (CLEAR NPT).
The initial search for pilot RCTs yielded 3857 titles, of which 49 articles were relevant for this review. 73.5% (36/49) of the orthopaedic pilot RCTs were framed as feasibility trials. Of these, 5 corresponding definitive trials (10.2%) were found, of which four were published and one ongoing. Based on author responses, the lack of a definitive RCT following the pilot trial was attributed to a lack of funding, inadequacies in recruitment, and belief that the pilot RCT sufficiently answered the research question.
Based on this systematic review, most pilot RCTs were characterized as feasibility trials. However, the majority of published pilot RCTs did not lead to definitive trials. This discrepancy was mainly attributed to poor feasibility (e.g. poor recruitment) and lack of funding for an orthopaedic surgical definitive trial. In recent years this discrepancy may be due to researchers saving on time and cost by rolling their pilot patients into the definitive RCT rather than publish a separate pilot trial.
本系统评价的主要目的是研究骨科手术文献中初步随机对照试验（RCT）的特征，包括界定为可行性试验的比例以及那些导致确定性RCT的试验。本评价旨在回答初步RCT是否能导致确定性RCT的问题，同时调查骨科初步试验的质量、可行性和总体发表趋势。
从三个电子数据库（EMBASE、MEDLINE和Pubmed）中检索骨科文献中的初步RCT，检索时间从数据库建立至2018年1月。检索标准包括对至少一种骨科手术干预的评估、对人类的研究以及英文发表。两名评价者独立筛选初步试验库，并搜索相应的确定性试验。对筛选出的初步RCT进行评估，以确定与涉及手术干预的大规模临床试验的效率、成本和/或及时性相关的可行性结果。使用《非药物试验报告评估清单》（CLEAR NPT）评估初步试验和确定性试验的质量。
初步检索初步RCT得到3857个标题，其中49篇文章与本评价相关。73.5%（36/49）的骨科初步RCT被界定为可行性试验。其中，找到了5个相应的确定性试验（10.2%），其中4个已发表，1个正在进行。根据作者的回复，初步试验后缺乏确定性RCT归因于资金不足、招募不足以及认为初步RCT已充分回答了研究问题。
基于本系统评价，大多数初步RCT的特征为可行性试验。然而，大多数已发表的初步RCT并未导致确定性试验。这种差异主要归因于可行性差（如招募困难）以及缺乏骨科手术确定性试验的资金。近年来，这种差异可能是由于研究人员通过将初步试验患者纳入确定性RCT而节省时间和成本，而不是发表单独的初步试验。
