Hammersmith Functional Motor Scale and Motor Function Measure-20 in non ambulant SMA patients |
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| Affiliation: | 1. Department of Paediatric Neurology, Catholic University, Rome, Italy;2. Dubowitz Neuromuscular Centre, Institute of Child Health, University College, London, UK;3. Child Neurology, University Hospitals Leuven, Leuven, Belgium;4. Department of Neurology, Academic Medical Centre, University of Amsterdam, Amsterdam, The Netherlands;5. Service of Physical Medicine and Rehabilitation, University Hospital Sant Joan de Deu, Barcelona, Spain;6. University Medical Centre, Freiburg, Germany;7. Child and Adolescence Neuropsychiatry Unit, Department of Neuroscience, and Department of Laboratory Medicine, Unit of Molecular Medicine, Bambino Gesù Hospital, Rome, Italy;8. Institute of Genetic Medicine, Newcastle Upon Tyne, UK;9. Institute of Myology, Groupe hospitalier La Pitié Salpétrière, Paris, France;1. Neuromuscular Diseases Unit, Department of Neurology, Tel Aviv Sourasky Medical Center, Tel Aviv, Israel;2. Sackler Faculty of Medicine, Tel-Aviv University, Tel-Aviv, Israel;3. Lunenfeld Tanenbaum Research Institute, Sinai Health System, University of Toronto, Toronto, Canada;4. Ellen and Martin Prosserman Centre for Neuromuscular Diseases, Division of Neurology, Department of Medicine, University Health Network, University of Toronto, Toronto, Canada;1. Institute of Microbial Chemistry (BIKAKEN), Tokyo, 3-14-23 Kamiosaki, Shinagawa-ku, Tokyo 141-0021, Japan;2. Institute of Medical Genetics, Tokyo Women’s Medical University, 10-22 Kawadacho, Shinjyuku-ku, Tokyo 162-0054, Japan;3. Merck Millipore Merck Ltd., Arco Tower 5F, 1-8-1 Shimomeguro, Megro-ku, Tokyo 153-8927, Japan;1. Child Neurology and Psychiatry Unit, Rome, Italy;2. Unit of Neuromuscular and Neurodegenerative Diseases, Department of Neurosciences, Bambino Gesù Childrens Hospital, Rome, Italy;3. Department of Neurosciences, Psychiatry and Anaesthesiology, University of Messina, Italy;4. Department of Experimental Medicine, University of Naples, Italy;5. Department of Developmental Neuroscience, Stella Maris Institute, Italy;6. Neuromuscular Disease Unit, G. Gaslini Institute, Genoa, Italy;7. Neuromuscular Center, SG. Battista Hospital, University of Turin, Italy;8. Department of Neurosciences, University of Padua, Italy;9. IRCCS “C. Mondino” Foundation, Pavia, Italy;10. IRCCS Eugenio Medea, Bosisio Parini, Italy;11. Child Neurology and Psychiatry Unit, IRCCS Institute of Neurological Sciences, Bellaria Hospital, Bologna, Italy;12. Centro Clinico Nemo, Milan, Italy;13. Pediatric Neurology and Neuroradiology Units, Neurological Institute C. Besta, Milan, Italy;14. Biostatistics Unit, Department of Health Sciences, University of Genoa, Italy;1. Department of Pediatrics, MetroHealth Medical Center, Case Western Reserve University, Cleveland, OH, USA;2. John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK;3. RTI International, Research Triangle Park, NC, USA;4. Department of Rehabilitation Medicine, Seattle Children''s Hospital, Seattle, WA, USA;5. Section of Pediatric Gastroenterology, Hepatology, and Nutrition, Children''s Hospital Colorado, Aurora, CO, USA;6. Doctor of Physical Therapy Division, Department of Orthopaedics, Duke University School of Medicine, Durham, NC, USA;7. Department of Neurology, University of Pittsburgh School of Medicine, and Neurology Service, Department of Veterans Affairs Medical Center, Pittsburgh, PA, USA;8. Division of Endocrinology and Metabolism, Children''s Hospital of Eastern Ontario, and University of Ottawa, Ottawa, ON, Canada;9. School of Medicine and Dentistry, University of Rochester, Rochester, NY, USA;10. Rare Disorders and Health Outcomes Team, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA;11. Medical Nutrition Consulting of Media LLC, and Children''s Hospital of Philadelphia, Philadelphia, PA, USA;12. Center for Genetic Muscle Disorders, Kennedy Krieger Institute, and Johns Hopkins School of Medicine, Baltimore, MD, USA;13. Division of Endocrinology and Diabetes, Golisano Children''s Hospital, University of Rochester Medical Center, Rochester, NY, USA;1. Department of Neuropaediatrics and Muscle Disorders, University Medical Center, Freiburg, Germany;2. Institute of Medical Biometry and Medical Informatics, University of Freiburg, Germany;3. Department of Neuropaediatrics, Charité University Medical School, Berlin, Germany;4. Department of Paediatric Neurology, University Hospital Essen, Germany;5. Department of Paediatric Endocinology, University Hospital Essen, Germany;6. Department of Paediatric Neurology and Developmental Medicine, Dr. Von Hauner Children’s Hospital, München, Germany;7. Department of Paediatrics and Paediatric Neurology, Georg August University, Göttingen, Germany |
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| Abstract: | The aim of this prospective longitudinal multi centric study was to evaluate the correlation between the Hammersmith Functional Motor Scale and the 20 item version of the Motor Function Measure in non ambulant SMA children and adults at baseline and over a 12 month period. Seventy-four non-ambulant patients performed both measures at baseline and 49 also had an assessment 12 month later. At baseline the scores ranged between 0 and 40 on the Hammersmith Motor function Scale and between 3 and 45 on the Motor Function Measure 20. The correlation between the two scales was 0.733. The 12 month changes ranged between −11 and 4 for the Hammersmith and between −11 and 7 for the Motor Function Measure 20. The correlation between changes was 0.48. Our results suggest that both scales provide useful information although they appeared to work differently at the two extremes of the spectrum of abilities. The Hammersmith Motor Function Scale appeared to be more suitable in strong non ambulant patients, while the Motor Function Measures appeared to be more sensitive to capture activities and possible changes in the very weak patients, including more items capturing axial and upper limb activities. The choice of these measures in clinical trials should therefore depend on inclusion criteria and magnitude of expected changes. |
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| Keywords: | Spinal muscular atrophy Outcome measures |
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