Different trajectories in upper limb and gross motor function in spinal muscular atrophy

Giorgia Coratti, Maria Carmela Pera, Jacqueline Montes, Amy Pasternak, Mariacristina Scoto, Giovanni Baranello, Sonia Messina, Sally Dunaway Young, Allan M. Glanzman, Tina Duong, Roberto De Sanctis, Elena Stacy Mazzone, Evelin Milev, Annemarie Rohwer, Matthew Civitello, Marika Pane, Laura Antonaci, Anna Lia Frongia, Maria Sframeli, Gian Luca VitaAdele DʼAmico, Adele D'Amico, Irene Mizzoni, Emilio Albamonte, Basil T. Darras, Enrico Bertini, Enrico Silvio Bertini, Valeria A. Sansone, Francesca Bovis, John Day, Claudio Bruno, Francesco Muntoni, Darryl C. De Vivo, Richard Finkel, Eugenio Maria Mercuri

Risultato della ricerca: Contributo in rivistaArticolo in rivista


Introduction: 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. Methods: 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). Results: 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. Discussion: 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.
Lingua originaleEnglish
pagine (da-a)552-559
Numero di pagine8
Stato di pubblicazionePubblicato - 2021


  • disease severity
  • motor
  • neuromuscular disorders
  • outcome measures
  • spinal muscular atrophy


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