標題: Reliability and Validity of Outcome Measures of In-Hospital and At-Home Visits in a Randomized, Double-Blind, Placebo-Controlled Trial for Spinal Muscular Atrophy
作者: Chen, Tai-Heng
Yang, Yi-Hsin
Mai, Hui-Hsia
Liang, Wen-Chen
Wu, Yi-Ching
Wang, Hui-Yi
Jong, Yuh-Jyh
生物科技學系
Department of Biological Science and Technology
關鍵字: spinal muscular atrophy;clinical trial;outcome measures;reliability and validity
公開日期: 1-Dec-2014
摘要: 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.
URI: http://dx.doi.org/10.1177/0883073813506935
http://hdl.handle.net/11536/124088
ISSN: 0883-0738
DOI: 10.1177/0883073813506935
期刊: JOURNAL OF CHILD NEUROLOGY
Volume: 29
Issue: 12
起始頁: 1680
結束頁: 1684
Appears in Collections:Articles