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European Cross-Sectional Survey of Current Care Practices for Duchenne Muscular Dystrophy Reveals Regional and Age-Dependent Differences

J. Vry, K. Gramsch, S. Rodger, R. Thompson, BF. Steffensen, J. Rahbek, S. Doerken, A. Tassoni, ML. Beytía, V. Guergueltcheva, T. Chamova, I. Tournev, A. Kostera-Pruszczyk, A. Kaminska, A. Lusakowska, L. Mrazova, L. Pavlovska, J. Strenkova, P....

. 2016 ; 3 (4) : 517-527.

Language English Country Netherlands

Document type Journal Article, Research Support, Non-U.S. Gov't

Persistent link   https://www.medvik.cz/link/bmc18010910

BACKGROUND: Publication of comprehensive clinical care guidelines for Duchenne muscular dystrophy (DMD) in 2010 was a milestone for DMD patient management. Our CARE-NMD survey investigates the neuromuscular, medical, and psychosocial care of DMD patients in Europe, and compares it to the guidelines. METHODS: A cross-sectional survey of 1677 patients contacted via the TREAT-NMD patient registries was conducted using self-report questionnaires in seven European countries. RESULTS: Survey respondents were 861 children and 201 adults. Data describe a European DMD population with mean age of 13.0 years (range 0.8-46.2) of whom 53% had lost ambulation (at 10.3 years of age, median). Corticosteroid medication raised the median age for ambulatory loss from 10.1 years in patients never medicated to 11.4 years in patients who received steroids (p < 0.0001). The majority of patients reported receiving care in line with guidelines, although we identified significant differences between countries and important shortcomings in prevention and treatment. Summarised, 35% of patients aged≥ nine years received no corticosteroid medication, 24% of all patients received no regular physiotherapy, echocardiograms were not performed regularly in 22% of patients, pulmonary function was not regularly assessed in 71% of non-ambulatory patients. Patients with regular follow-up by neuromuscular specialists were more likely to receive care according to guidelines, were better satisfied, and experienced shorter unplanned hospitalization periods.

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$a Vry, Julia $u Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Freiburg, Germany.
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$a BACKGROUND: Publication of comprehensive clinical care guidelines for Duchenne muscular dystrophy (DMD) in 2010 was a milestone for DMD patient management. Our CARE-NMD survey investigates the neuromuscular, medical, and psychosocial care of DMD patients in Europe, and compares it to the guidelines. METHODS: A cross-sectional survey of 1677 patients contacted via the TREAT-NMD patient registries was conducted using self-report questionnaires in seven European countries. RESULTS: Survey respondents were 861 children and 201 adults. Data describe a European DMD population with mean age of 13.0 years (range 0.8-46.2) of whom 53% had lost ambulation (at 10.3 years of age, median). Corticosteroid medication raised the median age for ambulatory loss from 10.1 years in patients never medicated to 11.4 years in patients who received steroids (p < 0.0001). The majority of patients reported receiving care in line with guidelines, although we identified significant differences between countries and important shortcomings in prevention and treatment. Summarised, 35% of patients aged≥ nine years received no corticosteroid medication, 24% of all patients received no regular physiotherapy, echocardiograms were not performed regularly in 22% of patients, pulmonary function was not regularly assessed in 71% of non-ambulatory patients. Patients with regular follow-up by neuromuscular specialists were more likely to receive care according to guidelines, were better satisfied, and experienced shorter unplanned hospitalization periods.
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$a Gramsch, Kathrin $u Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Freiburg, Germany.
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$a Rodger, Sunil $u The John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, International Centre for Life, Central Parkway, Newcastle upon Tyne, UK.
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$a Thompson, Rachel $u The John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, International Centre for Life, Central Parkway, Newcastle upon Tyne, UK.
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$a Steffensen, Birgit F $u National Rehabilitation Center for Neuromuscular Diseases, Aarhus, Denmark.
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$a Rahbek, Jes $u National Rehabilitation Center for Neuromuscular Diseases, Aarhus, Denmark.
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$a Doerken, Sam $u Institute for Medical Biometry and Statistics, Faculty of Medicine and Medical Center - University of Freiburg, Freiburg, Germany.
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$a Tassoni, Adrian $u Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Freiburg, Germany.
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$a Beytía, María de Los Angeles $u Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Freiburg, Germany.
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$a Guergueltcheva, Velina $u Medical University of Sofia, Department of Neurology, University Hospital Sofiamed and Bulgarian Neuromuscular Disorders Society, Sofia, Bulgaria.
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$a Chamova, Teodora $u Medical University of Sofia, Department of Neurology, University Hospital Alexandrovska and Bulgarian Neuromuscular Disorders Society, Sofia, Bulgaria.
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$a Tournev, Ivailo $u Medical University of Sofia, Department of Neurology, University Hospital Alexandrovska and Bulgarian Neuromuscular Disorders Society, Sofia, Bulgaria.
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$a Kostera-Pruszczyk, Anna $u Department of Neurology, Medical University of Warsaw, Warsaw, Poland.
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$a Kaminska, Anna $u Department of Neurology, Medical University of Warsaw, Warsaw, Poland.
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$a Lusakowska, Anna $u Department of Neurology, Medical University of Warsaw, Warsaw, Poland.
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$a Mrazova, Lenka $u Department of Paediatric Neurology, University Hospital in Brno and Faculty of Medicine, Masaryk University Brno, Czech Republic.
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$a Pavlovska, Lenka $u Institute of Biostatistics and Analyses, Masaryk University Brno, Czech Republic.
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$a Strenkova, Jana $u Institute of Biostatistics and Analyses, Masaryk University Brno, Czech Republic.
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$a Vondráček, Petr $u Department of Paediatric Neurology, University Hospital in Brno and Faculty of Medicine, Masaryk University Brno, Czech Republic.
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$a Garami, Marta $u Department of Molecular Genetics and Diagnostics, National Institute of Environmental Health, Budapest, Hungary.
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$a Karcagi, Veronika $u Department of Molecular Genetics and Diagnostics, National Institute of Environmental Health, Budapest, Hungary.
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$a Herczegfalvi, Ágnes $u Department of Pediatrics, Semmelweis University, Budapest, Hungary.
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$a Bushby, Katherine $u The John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, International Centre for Life, Central Parkway, Newcastle upon Tyne, UK.
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$a Lochmüller, Hanns $u The John Walton Muscular Dystrophy Research Centre and MRC Centre for Neuromuscular Diseases, Institute of Genetic Medicine, Newcastle University, International Centre for Life, Central Parkway, Newcastle upon Tyne, UK.
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$a Kirschner, Janbernd $u Department of Neuropediatrics and Muscle Disorders, Medical Center - University of Freiburg, Freiburg, Germany.
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