title: 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.
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.
authors.affiliation: 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.
authors.forename: Tai-Heng
authors.initials: TH
authors.lastname: Chen
authors.affiliation: Statistical Analysis Laboratory, Department of Medical Research, Kaohsiung Medical University Hospital, Kaohsiung, Taiwan.
authors.forename: Yi-Hsin
authors.initials: YH
authors.lastname: Yang
authors.affiliation: Department of Pediatrics, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan.
authors.forename: Hui-Hsia
authors.initials: HH
authors.lastname: Mai
authors.affiliation: Department of Pediatrics, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan.
authors.forename: Wen-Chen
authors.initials: WC
authors.lastname: Liang
authors.affiliation: Department of Physical Therapy, College of Health Science, Kaohsiung Medical University, Kaohsiung, Taiwan.
authors.forename: Yi-Ching
authors.initials: YC
authors.lastname: Wu
authors.affiliation: Department of Physical Therapy, College of Health Science, Kaohsiung Medical University, Kaohsiung, Taiwan.
authors.forename: Hui-Yi
authors.initials: HY
authors.lastname: Wang
authors.affiliation: Graduate Institute of Medicine, College of Medicine, Kaohsiung Medical University, Kaohsiung, Taiwan Department of Pediatrics, Kaohsiung Medical University Hospital, Kaohsiung Medical University, Kaohsiung, Taiwan Department of Laboratory Medicine, Kaohsiung Medical University Hospital, Kaohsiung, Taiwan Department of Biological Science and Technology, National Chiao Tung University, Hsinchu, Taiwan yjjong@kmu.edu.tw.
authors.forename: Yuh-Jyh
authors.initials: YJ
authors.lastname: Jong
mesh_terms: D000328:Adult; D002648:Child; D004185:Disability Evaluation; D004311:Double-Blind Method; D005260:Female; D006739:Hospital Administration; D006792:House Calls; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D017063:Outcome Assessment, Health Care / Q000379:methods*; D015203:Reproducibility of Results; D012189:Retrospective Studies; D012720:Severity of Illness Index; D014897:Spinal Muscular Atrophies of Childhood / Q000628:therapy*; D013223:Statistics as Topic; D016896:Treatment Outcome; D055815:Young Adult
keywords: clinical trial; outcome measures; reliability and validity; spinal muscular atrophy