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

Tai Heng Chen, Yi-Hsin Yang, Hui-Hsia Mai, Wen-Liang Chen, Yi Ching Wu, Ying Hui Wang, Yuh-Jyh Jong

Research output: Contribution to journalArticle

5 Scopus citations

Abstract

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.
Original languageEnglish
Pages (from-to)1680-1684
Number of pages5
JournalJournal of Child Neurology
Volume29
Issue number12
DOIs
StatePublished - Dec 2014

Keywords

  • spinal muscular atrophy; clinical trial; outcome measures; reliability and validity

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