Chen Tai-Heng, Yang Yi-Hsin, Mai Hui-Hsia, Liang Wen-Chen, Wu Yi-Ching, Wang Hui-Yi, Jong Yuh-Jyh
Division of Pediatric Emergency, Department of Emergency, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan Graduate Institute of Medicine, College of Medicine, Kaohsiung Medical University, Kaohsiung, Taiwan.
Statistical Analysis Laboratory, Department of Medical Research, Kaohsiung Medical University Hospital, Kaohsiung, Taiwan.
J Child Neurol. 2014 Dec;29(12):1680-4. doi: 10.1177/0883073813506935. Epub 2013 Oct 24.
We used at-home assessments in a clinical trial to relieve the visit burden for participants. A total of 57 patients with type II or III spinal muscular atrophy were enrolled and 10 of them (7 type II and 3 type III) received at-home assessments. The primary end points were Gross Motor Function Measure, Manual Muscle Test, and serum biomarker. The secondary endpoints were Modified Hammersmith Functional Motor Scale and forced vital capacity. The correlation coefficients and analysis of covariance showed good reliability and validity of all outcome measures. Except for Gross Motor Function Measure and Modified Hammersmith Functional Motor Scale, there were no significant differences in measures between in-hospital and at-home groups (intersubject) or among 3 patients who received both at-home and in-hospital visits (intrasubject). We concluded that at-home assessments could provide sufficient reliability in a controlled trial. This modification could help design a successful clinical trial for spinal muscular atrophy.
我们在一项临床试验中采用居家评估，以减轻参与者的就诊负担。总共招募了57例II型或III型脊髓性肌萎缩症患者，其中10例（7例II型和3例III型）接受了居家评估。主要终点指标为粗大运动功能测量、徒手肌力测试和血清生物标志物。次要终点指标为改良哈默史密斯功能运动量表和用力肺活量。相关系数和协方差分析表明，所有结局指标均具有良好的信度和效度。除粗大运动功能测量和改良哈默史密斯功能运动量表外，住院组和居家组（受试者间）或接受过居家和住院就诊的3例患者（受试者内）之间的测量指标无显著差异。我们得出结论，居家评估在对照试验中可提供足够的可靠性。这种改进有助于设计一项成功的脊髓性肌萎缩症临床试验。
