Different trajectories in upper limb and gross motor function in spinal muscular atrophy.
disease severity
motor
neuromuscular disorders
outcome measures
spinal muscular atrophy
Journal
Muscle & nerve
ISSN: 1097-4598
Titre abrégé: Muscle Nerve
Pays: United States
ID NLM: 7803146
Informations de publication
Date de publication:
11 2021
11 2021
Historique:
revised:
20
07
2021
received:
31
01
2021
accepted:
24
07
2021
pubmed:
31
7
2021
medline:
18
3
2022
entrez:
30
7
2021
Statut:
ppublish
Résumé
The Hammersmith Functional Motor Scale Expanded (HFMSE) and the Revised Upper Limb Module (RULM) have been widely used in natural history studies and clinical trials. Our aim was to establish how the scales relate to each other at different age points in spinal muscular atrophy (SMA) type 2 and 3, and to describe their coherence over 12 mo. The study was performed by cross-sectional and longitudinal reanalysis of previously published natural history data. The longitudinal analysis of the 12-mo changes also included the analysis of concordance between scales with changes grouped as stable (±2 points), improved (>+2) or declined (>-2). Three hundred sixty-four patients were included in the cross-sectional analysis, showing different trends in score and point of slope change for the two scales. For type 2, the point of slope change was 4.1 y for the HFMSE and 5.8 for the RULM, while for type 3, it was 6 y for the HFMSE and 7.3 for the RULM. One-hundred-twenty-one patients had at least two assessments at 12 mo. Full concordance was found in 57.3% of the assessments, and in 40.4% one scale remained stable and the other changed. Each scale appeared to be more sensitive to specific age or functional subgroups. The two scales, when used in combination, may increase the sensitivity to detect clinically meaningful changes in motor function in patients with SMA types 2 and 3.
Identifiants
pubmed: 34327716
doi: 10.1002/mus.27384
pmc: PMC9291175
doi:
Substances chimiques
Oligonucleotides
0
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
552-559Commentaires et corrections
Type : CommentIn
Informations de copyright
© 2021 The Authors. Muscle & Nerve published by Wiley Periodicals LLC.
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