Carole Vuillerot1, Katherine G Meilleur2, Minal Jain3, Melissa Waite3, Tianxia Wu4, Melody Linton2, Jahannaz Datsgir4, Sandra Donkervoort4, Meganne E Leach5, Anne Rutkowski6, Pascal Rippert7, Christine Payan8, Jean Iwaz9, Dalil Hamroun10, Carole Bérard11, Isabelle Poirot11, Carsten G Bönnemann12. 1. Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Hospices Civils de Lyon, L'Escale, Pediatric Physical Medicine and Rehabilitation Department, Bron, France; Université de Lyon, Lyon, France; CNRS UMR 5558, Laboratoire de Biométrie et Biologie Evolutive, Equipe Biostatistique Santé, Pierre-Bénite, France. Electronic address: carole.vuillerot@chu-lyon.fr. 2. National Institute of Nursing Research, National Institutes of Health, Bethesda, MD. 3. Mark O. Hatfield Clinical Research Center, National Institutes of Health, Bethesda, MD. 4. Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD. 5. Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Children's National Health System, Washington, DC. 6. Cure Congenital Muscular Dystrophy and Kaiser Southern California Permanente Medical Group, Los Angeles, CA. 7. Hospices Civils de Lyon, L'Escale, Pediatric Physical Medicine and Rehabilitation Department, Bron, France; Hospices Civils de Lyon, Pôle Information Médicale Évaluation Recherche, Lyon, France. 8. Assistance Publique-Hôpitaux de Paris, Hôpital Pitié-Salpétrière, Department of Clinical Pharmacology, Paris, France. 9. Université de Lyon, Lyon, France; CNRS UMR 5558, Laboratoire de Biométrie et Biologie Evolutive, Equipe Biostatistique Santé, Pierre-Bénite, France; Hospices Civils de Lyon, Service de Biostatistique, Lyon, France. 10. Centre Hospitalo-Universitaire de Montpellier, Montpellier, France. 11. Hospices Civils de Lyon, L'Escale, Pediatric Physical Medicine and Rehabilitation Department, Bron, France. 12. Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD. Electronic address: carsten.bonnemann@nih.gov.
Abstract
OBJECTIVE: To develop and validate an English version of the Neuromuscular (NM)-Score, a classification for patients with NM diseases in each of the 3 motor function domains: D1, standing and transfers; D2, axial and proximal motor function; and D3, distal motor function. DESIGN: Validation survey. SETTING: Patients seen at a medical research center between June and September 2013. PARTICIPANTS: Consecutive patients (N=42) aged 5 to 19 years with a confirmed or suspected diagnosis of congenital muscular dystrophy. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: An English version of the NM-Score was developed by a 9-person expert panel that assessed its content validity and semantic equivalence. Its concurrent validity was tested against criterion standards (Brooke Scale, Motor Function Measure [MFM], activity limitations for patients with upper and/or lower limb impairments [ACTIVLIM], Jebsen Test, and myometry measurements). Informant agreement between patient/caregiver (P/C)-reported and medical doctor (MD)-reported NM scores was measured by weighted kappa. RESULTS: Significant correlation coefficients were found between NM scores and criterion standards. The highest correlations were found between NM-score D1 and MFM score D1 (ρ=-.944, P<.0001), ACTIVLIM (ρ=-.895, P<.0001), and hip abduction strength by myometry (ρ=-.811, P<.0001). Informant agreement between P/C-reported and MD-reported NM scores was high for D1 (κ=.801; 95% confidence interval [CI], .701-.914) but moderate for D2 (κ=.592; 95% CI, .412-.773) and D3 (κ=.485; 95% CI, .290-.680). Correlation coefficients between the NM scores and the criterion standards did not significantly differ between P/C-reported and MD-reported NM scores. CONCLUSIONS: Patients and physicians completed the English NM-Score easily and accurately. The English version is a reliable and valid instrument that can be used in clinical practice and research to describe the functional abilities of patients with NM diseases.
OBJECTIVE: To develop and validate an English version of the Neuromuscular (NM)-Score, a classification for patients with NM diseases in each of the 3 motor function domains: D1, standing and transfers; D2, axial and proximal motor function; and D3, distal motor function. DESIGN: Validation survey. SETTING:Patients seen at a medical research center between June and September 2013. PARTICIPANTS: Consecutive patients (N=42) aged 5 to 19 years with a confirmed or suspected diagnosis of congenital muscular dystrophy. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: An English version of the NM-Score was developed by a 9-person expert panel that assessed its content validity and semantic equivalence. Its concurrent validity was tested against criterion standards (Brooke Scale, Motor Function Measure [MFM], activity limitations for patients with upper and/or lower limb impairments [ACTIVLIM], Jebsen Test, and myometry measurements). Informant agreement between patient/caregiver (P/C)-reported and medical doctor (MD)-reported NM scores was measured by weighted kappa. RESULTS: Significant correlation coefficients were found between NM scores and criterion standards. The highest correlations were found between NM-score D1 and MFM score D1 (ρ=-.944, P<.0001), ACTIVLIM (ρ=-.895, P<.0001), and hip abduction strength by myometry (ρ=-.811, P<.0001). Informant agreement between P/C-reported and MD-reported NM scores was high for D1 (κ=.801; 95% confidence interval [CI], .701-.914) but moderate for D2 (κ=.592; 95% CI, .412-.773) and D3 (κ=.485; 95% CI, .290-.680). Correlation coefficients between the NM scores and the criterion standards did not significantly differ between P/C-reported and MD-reported NM scores. CONCLUSIONS:Patients and physicians completed the English NM-Score easily and accurately. The English version is a reliable and valid instrument that can be used in clinical practice and research to describe the functional abilities of patients with NM diseases.
Authors: Laure Vandervelde; Peter Y K Van den Bergh; Nathalie Goemans; Jean-Louis Thonnard Journal: Neuromuscul Disord Date: 2007-04-11 Impact factor: 4.296
Authors: C Vuillerot; P Rippert; S Roche; C Bérard; F Margirier; C de Lattre; I Poirot; A Berruyer; V Tiffreau; M Fournier-Mehouas; F Bouhour; J-A Urtizberea; A Renders; R Ecochard Journal: Ann Phys Rehabil Med Date: 2013-10-18
Authors: M H Brooke; G M Fenichel; R C Griggs; J R Mendell; R Moxley; J P Miller; M A Province Journal: Muscle Nerve Date: 1983-02 Impact factor: 3.217