Muscle & nerve · 2015
Spinal muscular atrophy functional composite score: A functional measure in spinal muscular atrophy.
СтатьяНазвание и аннотация: Перевод пока не подготовлен
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Montes Jacqueline, Glanzman Allan M, Mazzone Elena S, Martens William B, Dunaway Sally, Pasternak Amy, Riley Susan O, Quigley Janet, Pandya Shree, De Vivo Darryl C, Kaufmann Petra, Chiriboga Claudia A, Finkel Richard S, Tennekoon Gihan I, Darras Basil T, Pane Marika, Mercuri Eugenio, Mcdermott Michael P, Pediatric Neuromuscular Clinical Research Network, Muscle Study Group, SMA Europe
Аннотация
INTRODUCTION: With clinical trials underway, our objective was to construct a composite score of global function that could 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 6-minute walk test as percent of 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.
CONCLUSIONS: This combination of measures broadens the spectrum of ability that can be quantified in type 2 and 3 SMA patients.
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 6-minute walk test as percent of 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.
CONCLUSIONS: This combination of measures broadens the spectrum of ability that can be quantified in type 2 and 3 SMA patients.