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Old measures and new scores in spinal muscular atrophy patients |
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| Authors: | Elena Mazzone PT Jacqueline Montes PT MA Marion Main MA Anna Mayhew PT PhD Danielle Ramsey PT Allan M. Glanzman PT DPT Sally Dunaway PT DPT Rachel Salazar PT Amy Pasternak PT Janet Quigley PT Marika Pane MD PhD Maria C. Pera MD Mariacristina Scoto MD Sonia Messina MD PhD Maria Sframeli MD Adele D'amico MD PhD Marleen Van Den Hauwe PT Serena Sivo MD Nathalie Goemans MD Basil T. Darras MD Petra Kaufmann MD MSc Enrico Bertini MD Darryl C. De Vivo MD Francesco Muntoni MD Richard Finkel MD Eugenio Mercuri MD PhD |
| Affiliation: | 1. Department of Paediatric Neurology, Catholic University, Rome, Italy;2. Department of Neurology, Columbia University Medical Center, New York, USA;3. Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, UK;4. Institute of Genetic Medicine, Newcastle University, Newcastle, UK;5. Department of Physical Therapy, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA;6. Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA;7. Department of Neurosciences, University of Messina, Messina, Italy;8. Unit of Neuromuscular and Neurodegenerative Disorders, Department of Neurosciences, IRCCS Bambino Gesù Children's Hospital, Rome, Italy;9. Department of Child Neurology, University Hospitals Leuven, Leuven, Belgium;10. Nemours Children's Hospital, University of Central Florida College of Medicine, Orlando, Florida, USA |
| Abstract: | 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. Muscle Nerve 52:435–437, 2015 |
| Keywords: | neuromuscular outcome measures Rasch analysis scoring spinal muscular atrophy |