Barnett Scott D, Heinemann Allen W, Libin Alexander, Houts Arthur C, Gassaway Julie, Sen-Gupta Sunil, Resch Aaron, Brossart Daniel F
Center of Excellence: Maximizing Rehabilitation Outcomes, James A. Haley Veterans Hospital, 8900 Grand Oak Circle, Tampa, FL 33612, USA.
J Rehabil Res Dev. 2012;49(1):175-86. doi: 10.1682/jrrd.2010.12.0242.
Rehabilitation research presents unique and challenging problems to investigators during both the design and analysis periods. Statistical issues regarding sample size requirements for an adequately powered study may be in direct conflict with realistic recruitment and subject retention goals. Issues of underpowered studies, sample size requirements, and recruitment goals plague rehabilitation research. Randomized clinical trials (RCTs) are typically narrow in scope and thus lack generalizability to everyday, yet specific, clinical problems; they are also costly and time-consuming and require large numbers of participants for randomization to have optimal, desired effects. Further, the RCT design may not be applicable to assistive technologies and environmental modifications-vital components of disability and rehabilitation research-nor is it appropriate in situations in which theoretical models of change are lacking or premature. Single-case designs are better suited for studies in which understanding and changing patient behavior and functional status are primary goals and the targeted sample sizes are less than 30 and frequently less than 10. Theoretical, methodological, and clinical reasons for using experimental and quasi-experimental single-case designs are presented. Recommendations for designing and conducting single-case studies that contribute to the evidence base are also discussed.
康复研究在设计和分析阶段都给研究者带来了独特且具有挑战性的问题。关于具备足够效力的研究所需样本量的统计问题,可能与现实的招募和受试者留存目标直接冲突。研究效力不足、样本量要求以及招募目标等问题困扰着康复研究。随机临床试验(RCT)通常范围较窄,因此缺乏对日常但具体的临床问题的普遍适用性;它们还成本高昂且耗时,并且需要大量参与者进行随机分组才能产生最佳的预期效果。此外,RCT设计可能不适用于辅助技术和环境改造——残疾与康复研究的重要组成部分——在缺乏或尚未成熟的变革理论模型的情况下也不合适。单病例设计更适合以理解和改变患者行为及功能状态为主要目标且目标样本量小于30且通常小于10的研究。本文介绍了使用实验性和准实验性单病例设计的理论、方法学和临床原因。还讨论了有助于建立证据基础的单病例研究设计与实施的建议。