Statistical and Methodological Considerations for Randomized Controlled Trial Design in Physical Medicine and Rehabilitation.
Statistical and Methodological Considerations for Randomized Controlled Trial Design in Physical Medicine and Rehabilitation.
复制标题
物理医学和康复中随机对照试验设计的统计和方法学考虑。
DOI：
10.1097/phm.0000000000002225
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发表时间：
2023
影响因子：
3
通讯作者：
Jain,NitinB
中科院分区：
文献类型：
--
作者：
Thakur,Bhaskar;Ayers,GregoryD;Atem,Folefac;DeClercq,JoshuaJ;Jain,NitinB
精心设计的评估物理医学和康复领域治疗的随机对照临床试验对于循证患者护理至关重要。然而，由于物理医学和康复领域的健康干预措施复杂，该领域的临床试验面临着独特的挑战。我们强调经常遇到的实证挑战，并就设计和进行随机对照试验的统计和方法学方法提供基于证据的建议。解决的一些问题包括康复环境中盲法治疗组的挑战、治疗方法的异质性、治疗效果的异质性、患者报告的结果测量的一致性以及不同信息规模对功效的影响。此外，我们还讨论了样本量和功效估计、适应治疗依从性差和缺失结果的挑战，以及纵向数据分析的首选统计方法。
Well-designed randomized controlled clinical trials assessing treatments in the field of physical medicine and rehabilitation are essential for evidence-based patient care. However, there are challenges unique to clinical trials in physical medicine and rehabilitation due to complex health interventions in this field. We highlight routinely encountered empirical challenges and provide evidence-based recommendations on statistical and methodological approaches for the design and conduct of randomized controlled trials. Some of the issues addressed include challenges with blinding treatment groups in a rehabilitation setting, heterogeneity in treatment therapy, heterogeneity of treatment effects, uniformity in patient-reported outcome measures, and effect on power with varying scales of information. Furthermore, we discuss challenges with estimation of sample size and power, adaption to poor compliance with treatment and missing outcomes, and preferred statistical approaches for longitudinal data analysis.