Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy
PLoS ONE, vol. 18, pp. e0283669
Abstract
The North Star ambulatory assessment (NSAA) is a functional motor outcome measure in Duchenne muscular dystrophy (DMD), widely used in clinical trials and natural history studies, as well as in clinical practice. However, little has been reported on the minimal clinically important difference (MCID) of the NSAA. The lack of established MCID estimates for NSAA presents challenges in interpreting the significance of the results of this outcome measure in clinical trials, natural history studies and clinical practice. Combining statistical approaches and patient perspectives, this study estimated MCID for NSAA using distribution-based estimates of 1/3 standard deviation (SD) and standard error of measurement (SEM), an anchor-based approach, with six-minute walk distance (6MWD) as the anchor, and evaluation of patient and parent perception using participant-tailored questionnaires. The MCID for NSAA in boys with DMD aged 7 to 10 years based on 1/3 SD ranged from 2.3-2.9 points, and that on SEM ranged from 2.9-3.5 points. Anchored on the 6MWD, the MCID for NSAA was estimated as 3.5 points. When the impact on functional abilities was considered using participant response questionnaires, patients and parent perceived a complete loss of function in a single item or deterioration of function in one to two items of the assessment as an important change. Our study examines MCID estimates for total NSAA scores using multiple approaches, including the impact of patient and parent perspective on within scale changes in items based on complete loss of function and deterioration of function, and provides new insight on evaluation of differences in these widely used outcome measure in DMD.
Authors 20
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Great Ormond Street Hospital · University College London
Affiliation as printed
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom
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Great Ormond Street Hospital · University College London
Affiliation as printed
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom
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Great Ormond Street Hospital · University College London
Affiliation as printed
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom
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NIHR Great Ormond Street Hospital Biomedical Research Centre · University College London
Affiliation as printed
NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom
Population, Policy & Practice Research and Teaching Department, UCL Great Ormond Street (GOS) Institute of Child Health, London, United Kingdom
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Great Ormond Street Hospital · University College London
Affiliation as printed
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom
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Great Ormond Street Hospital · University College London
Affiliation as printed
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom
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Great Ormond Street Hospital · University College London
Affiliation as printed
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom
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Great Ormond Street Hospital · University College London
Affiliation as printed
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom
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Great Ormond Street Hospital · University College London
Affiliation as printed
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom
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Newcastle upon Tyne Hospitals NHS Foundation Trust · Newcastle University
Affiliation as printed
The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom
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Università Cattolica del Sacro Cuore · Agostino Gemelli University Polyclinic
Affiliation as printed
Child Neurology Unite Centro Nemo, IRCCS Fondazione Policlinico Gemelli, Universita Cattolica del Sacro Cuore, Rome, Italy
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Great Ormond Street Hospital · NIHR Great Ormond Street Hospital Biomedical Research Centre · University College London
Affiliation as printed
NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom
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Affiliation as printed
Institute of Myology, Paris, France
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Leiden University Medical Center · ERN EURO-NMD
Affiliation as printed
Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands
European Reference Network for Rare Neuromuscular Diseases - ERN EURO NMD
European Reference Network for Rare Neuromuscular Diseases – ERN EURO NMD
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Radboud University Nijmegen · Radboud University Medical Center · Donders Centre for Neuroscience
Affiliation as printed
Department of Rehabilitation, Donders Center for Medical Neuroscience, Radboud University Medical Center, Nijmegen, The Netherlands
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University of Liège · University of Oxford · Institut de Myologie · Centre Hospitalier Universitaire de Liège · Centre hospitalier régional de la Citadelle
Affiliation as printed
Department of Paediatrics, MDUK Neuromuscular Center, University of Oxford, Oxford, United Kingdom
Division of Child Neurology, Reference Center for Neuromuscular Disease, Centre Hospitalier Régional de Références des Maladies Neuromusculaires, Department of Paediatrics, University Hospital Liège & University of La Citadelle, Liège, Belgium
Institute of Myology, Paris, France
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Newcastle upon Tyne Hospitals NHS Foundation Trust · Newcastle University
Affiliation as printed
The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom
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Università Cattolica del Sacro Cuore · Agostino Gemelli University Polyclinic
Affiliation as printed
Child Neurology Unit, Universita Cattolica del Sacro Cuore, Rome, Italy
Child Neurology Unite Centro Nemo, IRCCS Fondazione Policlinico Gemelli, Universita Cattolica del Sacro Cuore, Rome, Italy
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Great Ormond Street Hospital · University College London
Affiliation as printed
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom
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Francesco Muntoni A corresponding
Great Ormond Street Hospital · NIHR Great Ormond Street Hospital Biomedical Research Centre · University College London
Affiliation as printed
NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom
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