title: Upper limb evaluation and one-year follow up of non-ambulant patients with spinal muscular atrophy: an observational multicenter trial.
abstract: Assessment of the upper limb strength in non-ambulant neuromuscular patients remains challenging. Although potential outcome measures have been reported, longitudinal data demonstrating sensitivity to clinical evolution in spinal muscular atrophy patients are critically lacking. Our study recruited 23 non-ambulant patients, 16 patients (males/females = 6/10; median age 15.4 years with a range from 10.7 to 31.1 years) with spinal muscular atrophy type II and 7 patients (males/females = 2/5; median age 19.9 years with a range from 8.3 to 29.9 years) with type III. The Brooke functional score was on median 3 with a range from 2 to 6. The average total vital capacity was 46%, and seven patients required non-invasive ventilation at night. Patients were assessed at baseline, 6 months, and 1 year using the Motor Function Measure and innovative devices MyoGrip, MyoPinch, and MoviPlate, which assess handgrip strength, key pinch strength, and hand/finger extension-flexion function, respectively. The study demonstrated the feasibility and reliability of these measures for all patients, and sensitivity to negative changes after the age of 14 years. The younger patients showed an increase of the distal force in the follow-up period. The distal force measurements and function were correlated to different functional scales. These data represent an important step in the process of validating these devices as potential outcome measures for future clinical trials.
authors: Institute of Myology, Paris, France.
authors: Andreea Mihaela
authors: AM
authors: Seferian
authors: Institute of Myology, Paris, France.
authors: Amélie
authors: A
authors: Moraux
authors: Institute of Myology, Paris, France.
authors: Aurélie
authors: A
authors: Canal
authors: Institute of Myology, Paris, France.
authors: Valérie
authors: V
authors: Decostre
authors: Institute of Myology, Paris, France.
authors: Oumar
authors: O
authors: Diebate
authors: Institute of Myology, Paris, France; Department of Child Neurology, Centre Hospitalier Universitaire Amiens-Picardie, Amiens, France.
authors: Anne Gaëlle
authors: AG
authors: Le Moing
authors: Institute of Myology, Paris, France.
authors: Teresa
authors: T
authors: Gidaro
authors: Department of Pediatrics, Division of Pediatric Neurology and Metabolism, Neuromuscular Reference Center, Universitair Ziekenhuis Gent, Gent, Belgium.
authors: Nicolas
authors: N
authors: Deconinck
authors: Department of Pediatrics, Division of Pediatric Neurology and Metabolism, Neuromuscular Reference Center, Universitair Ziekenhuis Gent, Gent, Belgium.
authors: Frauke
authors: F
authors: Van Parys
authors: Department of Pediatrics, Division of Pediatric Neurology and Metabolism, Neuromuscular Reference Center, Universitair Ziekenhuis Gent, Gent, Belgium.
authors: Wendy
authors: W
authors: Vereecke
authors: Department of Pediatrics, Division of Pediatric Neurology and Metabolism, Neuromuscular Reference Center, Universitair Ziekenhuis Gent, Gent, Belgium.
authors: Sylvia
authors: S
authors: Wittevrongel
authors: Institute of Myology, Paris, France.
authors: Mélanie
authors: M
authors: Annoussamy
authors: Reference Center for Neuromuscular Disease, Assistance Publique-Hôpitaux de Paris-Hôpital Trousseau, Paris, France.
authors: Michèle
authors: M
authors: Mayer
authors: Reference Center for Neuromuscular Disease, Assistance Publique-Hôpitaux de Paris-Hôpital Trousseau, Paris, France.
authors: Kim
authors: K
authors: Maincent
authors: Department of Pediatrics, Centre Hospitalier Régional Universitaire de Lille-Hôpital Roger Salengro, Lille, France.
authors: Jean-Marie
authors: JM
authors: Cuisset
authors: Department of Physical Medicine and Rehabilitation, Centre Hospitalier Régional Universitaire de Lille-Hôpital Pierre Swynghedauw, Lille, France.
authors: Vincent
authors: V
authors: Tiffreau
authors: Reference Center for Neuromuscular Disease, Centre Hospitalier Régional de La Citadelle, Liège, Belgium.
authors: Severine
authors: S
authors: Denis
authors: Reference Center for Neuromuscular Disease, Centre Hospitalier Régional de La Citadelle, Liège, Belgium.
authors: Virginie
authors: V
authors: Jousten
authors: Department of Pediatrics, Centre de références Maladies Neuromusculaires Garches-Necker-Mondor-Hendaye and Endicap U1179 INSERM-Université de Versailles-Saint-Quentin-en-Yvelines, Assistance Publique-Hôpitaux de Paris-Hôpital Raymond-Poincaré, Garches, France.
authors: Susana
authors: S
authors: Quijano-Roy
authors: Institute of Myology, Paris, France; Thérapie des maladies du muscle strié / Institut de Myologie, Unité Mixte de Recherche S 974 Université Pierre et Marie Curie-Institut national de la santé et de la recherche médicale-Formation de Recherche en Evolution 3617 Centre national de la recherche scientifique-Association Institut de Myologie, Paris, France.
authors: Thomas
authors: T
authors: Voit
authors: Institute of Myology, Paris, France.
authors: Jean-Yves
authors: JY
authors: Hogrel
authors: Institute of Myology, Paris, France; Reference Center for Neuromuscular Disease, Centre Hospitalier Régional de La Citadelle, Liège, Belgium.
authors: Laurent
authors: L
authors: Servais
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D005260:Female; D005500:Follow-Up Studies; D018737:Hand Strength / Q000502:physiology; D006801:Humans; D008297:Male; D009134:Muscular Atrophy, Spinal / Q000503:physiopathology*; D063087:Noninvasive Ventilation; D052581:Pinch Strength / Q000502:physiology; D014897:Spinal Muscular Atrophies of Childhood / Q000503:physiopathology; D034941:Upper Extremity / Q000503:physiopathology*; D055815:Young Adult