Neuromuscular disorders : NMD · 2020
Age and baseline values predict 12 and 24-month functional changes in type 2 SMA.
СтатьяПоддержка исследования: вне правительства СШАНазвание и аннотация: Перевод в очереди
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Coratti Giorgia, Pera Maria C, Lucibello Simona, Montes Jacqueline, Pasternak Amy, Mayhew Anna, Glanzman Allan M, Young Sally Dunaway, Pane Marika, Scoto Mariacristina, Messina Sonia, Goemans Nathalie, Osorio Andres Nascimiento, Pedemonte Marina, Sansone Valeria, Bertini Enrico, De Vivo Darryl C, Finkel Richard, Muntoni Francesco, Mercuri Eugenio, ISMAC group and collaborators.
Аннотация
The aim of this retrospective study was to establish the range of functional changes at 12 and 24-month in 267 type 2 Spinal Muscular Atrophy (SMA) patients with multiple assessments. We included 652 Hammersmith Functional Motor Scale Expanded (HFMSE) assessments at 12 month- and 305 at 24 month- intervals. The cohort was subdivided by functional level, Survival of Motor Neuron copy number and age. Stable scores (± 2 points) were found in 68% at 12 months and in 55% at 24 months. A decrease ≥2 points was found in 21% at 12 months and in 35% at 24 months. An increase ≥2 points was found in 11% at 12 months and 9.5% at 24 months. The risk of losing ≥2 points increased with age and HFMSE score at baseline both at 12 and 24-month. For each additional HFMSE point at baseline, the relative risk of a >2 point decline at 12 months increases by 5% before age 5 years (p = 0.023), by 8% between 5 and 13 (p<0.001) and by 26% after 13 years (p = 0.003). The combination of age and HFMSE scores at baseline increased the ability to predict progression in type 2 SMA.