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

Abstract

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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@inproceedings{Vry2016EuropeanCS, title={European Cross-Sectional Survey of Current Care Practices for Duchenne Muscular Dystrophy Reveals Regional and Age-Dependent Differences}, author={Julia Vry and Kathrin Gramsch and Sunil Rodger and Rachel Thompson and Birgit Fynbo Steffensen and Jes Rahbek and Sam Doerken and Adrian Tassoni and Mar{\'i}a de los Angeles Beyt{\'i}a and Velina Guergueltcheva and Teodora Chamova and Ivailo Tournev and Anna Kostera-Pruszczyk and Anna Maria Kaminska and Anna Lusakowska and Lenka S Mrazova and Lenka Pavlovska and Jana Strenkov{\'a} and Petr Vondracek and Marta Garami and Veronika Karcagi and {\'A}gnes Herczegfalvi and Katherine Bushby and Hanns Lochm{\"{u}ller and Janbernd Kirschner}, booktitle={Journal of neuromuscular diseases}, year={2016} }