Audigé Laurent, Hanson Beate, Kopjar Branko
AO Clinical Investigation and Documentation, AO Center, Davos Platz, Switzerland.
Injury. 2006 Apr;37(4):340-8. doi: 10.1016/j.injury.2006.01.026. Epub 2006 Feb 17.
This paper discusses topics related to the planning and implementation of non-randomised clinical studies in orthopaedics. A well-conducted case-series is appropriate to demonstrate the safety of a surgical intervention. The case-series design involves the provision of a defined intervention to a group of patients with the ultimate objective of describing the final outcome, including such occurrences as complications. There is no alternative procedure serving as a control. The key aspects are to ensure enrolment of all eligible patients and to obtain a sufficiently large sample size to allow precise and valid estimation of complication risks. Targeted complications should be clearly defined and fully documented during a pre-defined follow-up period. Loss to follow-up should be minimised. Comparative studies are required to demonstrate treatment effectiveness. If a randomised controlled trial (RCT) is not feasible, an observational design such as a cohort or a case-control study should be considered. In observational designs, the treatment decision is made by the surgeons. In a case-control study, patients are selected based on their outcomes and their treatment or exposure status is recorded retrospectively. In a cohort study, groups of patients are selected based on their treatment and are followed for outcomes. There are numerous variations. Data can be collected prospectively or retrospectively; comparison groups may be concurrent or non-concurrent, or studied at different locations. The optimal design is tailored to clinical questions and research settings, while keeping in mind the respective methodological strengths and weaknesses of available options. The strength of the observational study is its proximity to daily clinical practice. The limitations are the possibility of numerous biases and confounding factors. Despite many challenges to the internal validity of non-randomised studies in orthopaedics surgery, it is possible to use such designs in order to provide reasonably valid answers to clinically important questions.
本文讨论了与骨科非随机临床研究的规划和实施相关的主题。精心开展的病例系列研究适合用于证明手术干预的安全性。病例系列设计涉及为一组患者提供特定干预，最终目的是描述最终结果，包括并发症等情况。没有替代程序作为对照。关键方面是确保纳入所有符合条件的患者，并获得足够大的样本量，以便准确有效地估计并发症风险。应在预先确定的随访期内明确界定并充分记录目标并发症。应尽量减少失访情况。需要进行比较研究来证明治疗效果。如果随机对照试验（RCT）不可行，则应考虑采用队列研究或病例对照研究等观察性设计。在观察性设计中，治疗决策由外科医生做出。在病例对照研究中，根据患者的结局选择患者，并回顾性记录他们的治疗或暴露情况。在队列研究中，根据患者的治疗情况选择患者组，并随访其结局。有许多变体。数据可以前瞻性或回顾性收集；比较组可以是同期或非同期的，或者在不同地点进行研究。最佳设计是根据临床问题和研究环境量身定制的，同时要牢记现有选项各自的方法学优势和劣势。观察性研究的优势在于其与日常临床实践接近。其局限性在于存在多种偏倚和混杂因素的可能性。尽管骨科手术中非随机研究的内部有效性面临诸多挑战，但仍有可能使用此类设计，以便为临床重要问题提供合理有效的答案。
