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Old measures and new scores in spinal muscular atrophy patients.
Mazzone, Elena; Montes, Jacqueline; Main, Marion; Mayhew, Anna; Ramsey, Danielle; Glanzman, Allan M; Dunaway, Sally; Salazar, Rachel; Pasternak, Amy; Quigley, Janet; Pane, Marika; Pera, Maria C; Scoto, Mariacristina; Messina, Sonia; Sframeli, Maria; D'amico, Adele; Van Den Hauwe, Marleen; Sivo, Serena; Goemans, Nathalie; Darras, Basil T; Kaufmann, Petra; Bertini, Enrico; De Vivo, Darryl C; Muntoni, Francesco; Finkel, Richard; Mercuri, Eugenio.
Afiliación
  • Mazzone E; Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
  • Montes J; Department of Neurology, Columbia University Medical Center, New York, USA.
  • Main M; Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, UK.
  • Mayhew A; Institute of Genetic Medicine, Newcastle University, Newcastle, UK.
  • Ramsey D; Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, UK.
  • Glanzman AM; Department of Physical Therapy, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
  • Dunaway S; Department of Neurology, Columbia University Medical Center, New York, USA.
  • Salazar R; Department of Neurology, Columbia University Medical Center, New York, USA.
  • Pasternak A; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
  • Quigley J; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
  • Pane M; Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
  • Pera MC; Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
  • Scoto M; Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, UK.
  • Messina S; Department of Neurosciences, University of Messina, Messina, Italy.
  • Sframeli M; Department of Neurosciences, University of Messina, Messina, Italy.
  • D'amico A; Unit of Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
  • Van Den Hauwe M; Department of Child Neurology, University Hospitals Leuven, Leuven, Belgium.
  • Sivo S; Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
  • Goemans N; Department of Child Neurology, University Hospitals Leuven, Leuven, Belgium.
  • Darras BT; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
  • Kaufmann P; Department of Neurology, Columbia University Medical Center, New York, USA.
  • Bertini E; Unit of Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, IRCCS Bambino Gesù Children's Hospital, Rome, Italy.
  • De Vivo DC; Department of Neurology, Columbia University Medical Center, New York, USA.
  • Muntoni F; Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, UK.
  • Finkel R; Nemours Children's Hospital, University of Central Florida College of Medicine, Orlando, Florida, USA.
  • Mercuri E; Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
Muscle Nerve ; 52(3): 435-7, 2015 Sep.
Article en En | MEDLINE | ID: mdl-26111847
INTRODUCTION: A recent Rasch analysis performed on the Hammersmith Functional Motor Scale-Expanded (HFMSE) in patients with spinal muscular atrophy (SMA) identified issues impacting scale validity, redundant items, and disordered thresholds on some items. METHODS: We modified the HMFSE scoring based on the Rasch analysis and on expert consensus to establish whether the traditional scoring overestimated the number of patients with changes within 2 points from baseline. Data were collected retrospectively from multicenter data sets in 255 type 2 and 3 SMA patients. RESULTS: The mean 12-month changes using the new and the traditional scoring system did not differ significantly (P > 0.05). The numbers of patients who improved or decreased by >2 points were also similar. CONCLUSIONS: The presence of outliers using the traditional scoring system was not due to overestimation of changes in activities that were tested bilaterally or to discrepancies in the scoring hierarchy of individual items.
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Índice de Severidad de la Enfermedad / Atrofias Musculares Espinales de la Infancia Tipo de estudio: Observational_studies / Prognostic_studies Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Male / Middle aged Idioma: En Revista: Muscle Nerve Año: 2015 Tipo del documento: Article País de afiliación: Italia
Texto completo
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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Índice de Severidad de la Enfermedad / Atrofias Musculares Espinales de la Infancia Tipo de estudio: Observational_studies / Prognostic_studies Límite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Male / Middle aged Idioma: En Revista: Muscle Nerve Año: 2015 Tipo del documento: Article País de afiliación: Italia