Age and baseline values predict 12 and 24-month functional changes in type 2 SMA

Giorgia Coratti, Maria Carmela Pera, Simona Lucibello, Jacqueline Montes, Amy Pasternak, Anna Mayhew, Allan M Glanzman, Sally Dunaway Young, Marika Pane, Mariacristina Scoto, Sonia Messina, Nathalie Goemans, Andres Nascimiento Osorio, Marina Pedemonte, Valeria Sansone, Enrico Bertini, Darryl C. De Vivo, Richard Finkel, Francesco Muntoni, Eugenio Maria Mercuri

Risultato della ricerca: Contributo in rivistaArticolo in rivista

Abstract

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.
Lingua originaleEnglish
pagine (da-a)756-764
Numero di pagine9
RivistaNeuromuscular Disorders
Volume30
DOI
Stato di pubblicazionePubblicato - 2020

Keywords

  • Hammersmith functional motor scale expanded
  • Neuromuscular disorders
  • Outcome measures
  • Spinal muscular atrophy

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