Title: | Spinal Muscular Atrophy functional composite score: A functional measure in spinal muscular atrophy. |
Journal : | Muscle & Nerve, 52, 6 |
Authors: | Montes J ; Glanzman AM ; Mazzone ES ; Martens WB ; Dunaway S ; Pasternak A ; Riley SO ; Quigley J ; Pandya S ; de Vivo DC ; Kaufmann P ; Chiriboga CA ; Finkel RS ; Tennekoon GI ; Darras BT ; Pane M ; Mercuri E ; McDermott MP |
Material Type: | Article |
Publication Date: | 12/2015 |
Size: | p. 942-7 |
Languages: | English |
Keywords : | cross-sectional study ; loongitudinal study |
Keywords: | SMA ; Spinal muscular atrophy ; clinical trial ; composite score ; motor function ; outcome measure |
Abstract: |
INTRODUCTION: With clinical trials underway, our objective was to construct a composite score of global function that will discriminate among people with Spinal Muscular Atrophy (SMA).
METHODS: Data were collected from 126 participants with SMA Types 2 and 3. Scores from the Hammersmith Functional Motor Scale-Expanded and Upper Limb Module were expressed as a percentage of the maximum score and Six-Minute Walk Test as percent predicted normal distance. A principal component analysis was performed on the correlation matrix for the 3 percentage scores. RESULTS: The first principal component yielded a composite score with approximately equal weighting of the 3 components and accounted for 82% of the total variability. The SMA Functional Composite score, an unweighted average of the 3 individual percentage scores, correlated almost perfectly with the first principal component. DISCUSSION: This combination of measures broadens the spectrum of ability that can be quantified in Type 2 and 3 SMA patients. This article is protected by copyright. All rights reserved. |
Pubmed / DOI : | DOI : 10.1002/mus.24670 / Pubmed : 25846132 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/25846132 |