title: Upper limb module in non-ambulant patients with spinal muscular atrophy: 12 month changes.
abstract: Recent studies have suggested that in non-ambulant patients affected by spinal muscular atrophy the Upper Limb Module can increase the range of activities assessed by the Hammersmith Functional Motor Scale Expanded. The aim of this study was to establish 12-month changes in the Upper Limb Module in a cohort of non-ambulant spinal muscular atrophy patients and their correlation with changes on the Hammersmith Functional Motor Scale Expanded. The Upper Limb Module scores ranged between 0 and 17 (mean 10.23, SD 4.81) at baseline and between 1 and 17 at 12 months (mean 10.27, SD 4.74). The Hammersmith Functional Motor Scale Expanded scores ranged between 0 and 34 (mean 12.43, SD 9.13) at baseline and between 0 and 34 at 12 months (mean 12.08, SD 9.21). The correlation betweeen the two scales was 0.65 at baseline and 0.72 on the 12 month changes. Our results confirm that the Upper Limb Module can capture functional changes in non-ambulant spinal muscular atrophy patients not otherwise captured by the other scale and that the combination of the two measures allows to capture changes in different subgroups of patients in whom baseline scores and functional changes may be influenced by several variables such as age.
authors.affiliation: Paediatric Neurology Unit, Catholic University, Rome, Italy.
authors.forename: Serena
authors.initials: S
authors.lastname: Sivo
authors.affiliation: Paediatric Neurology Unit, Catholic University, Rome, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Mazzone
authors.affiliation: Department of Paediatrics, Child Neurology and Psychiatry, Sapienza University, Rome, Italy.
authors.forename: Laura
authors.initials: L
authors.lastname: Antonaci
authors.affiliation: Paediatric Neurology Unit, Catholic University, Rome, Italy.
authors.forename: Roberto
authors.initials: R
authors.lastname: De Sanctis
authors.affiliation: Paediatric Neurology Unit, Catholic University, Rome, Italy.
authors.forename: Lavinia
authors.initials: L
authors.lastname: Fanelli
authors.affiliation: Paediatric Neurology Unit, Catholic University, Rome, Italy.
authors.forename: Concetta
authors.initials: C
authors.lastname: Palermo
authors.affiliation: Department of Neurology, Columbia University, New York, NY, United States.
authors.forename: Jacqueline
authors.initials: J
authors.lastname: Montes
authors.affiliation: Paediatric Neurology Unit, Catholic University, Rome, Italy.
authors.forename: Marika
authors.initials: M
authors.lastname: Pane
authors.affiliation: Paediatric Neurology Unit, Catholic University, Rome, Italy. Electronic address: mercuri@rm.unicatt.it.
authors.forename: Eugenio
authors.initials: E
authors.lastname: Mercuri
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D006801:Humans; D008137:Longitudinal Studies; D009134:Muscular Atrophy, Spinal / Q000235:genetics / Q000503:physiopathology*; D012720:Severity of Illness Index; D055533:Survival of Motor Neuron 1 Protein / Q000235:genetics; D013997:Time Factors; D034941:Upper Extremity / Q000503:physiopathology*; D055815:Young Adult
chemical_list: C526471:SMN1 protein, human; D055533:Survival of Motor Neuron 1 Protein
keywords: Hammersmith; Outcome measure; Spinal muscular atrophy; Upper limb