|Titre :||Spinal Muscular Atrophy functional composite score: A functional measure in spinal muscular atrophy.|
|Revue :||Muscle & Nerve, 52, 6|
|Auteurs :||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|
|Type de document :||Article|
|Année de publication :||12/2015|
|Format :||p. 942-7|
|Mots-clés :||étude longitudinale ; étude transversale|
|Mots-clés:||SMA ; Spinal muscular atrophy ; clinical trial ; composite score ; motor function ; outcome measure|
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|
|En ligne :||http://www.ncbi.nlm.nih.gov/pubmed/25846132|