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Compliance to Care Guidelines for Duchenne Muscular Dystrophy

Article type: Research Article

Authors: Landfeldt, Erik; | Lindgren, Peter | Bell, Christopher F. | Schmitt, Claude | Guglieri, Michela | Straub, Volker | Lochmüller, Hanns | Bushby, Katharine

Affiliations: OptumInsight, Stockholm, Sweden | Institute of Environmental Medicine, Karolinska Institutet, Stockholm, Sweden | Medical Management Centre, Department of Learning, Informatics, Management and Ethics, Karolinska Institutet, Stockholm, Sweden | GlaxoSmithKline, Research Triangle Park, North Carolina, USA | GlaxoSmithKline, Brentford, Middlesex, UK | Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK

Note: [] Equal contribution.

Note: [] Correspondence to: Professor Katharine Bushby, Institute of Genetic Medicine, International Centre for Life, Central Parkway, Newcastle upon Tyne, NE1 3BZ, UK. Tel.: +44 191 241 8757; E-mail: [email protected] Equal contribution.

Keywords: Muscular dystrophy, duchenne, practice guideline, delivery of health care, observational study

DOI: 10.3233/JND-140053

Journal: Journal of Neuromuscular Diseases, vol. 2, no. 1, pp. 63-72, 2015

Published: 2015
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Abstract

Background: International care guidelines for Duchenne muscular dystrophy (DMD) were published in 2010, but compliance in clinical practice is unknown. Objective: The objective of our study was to compare real-world DMD care in Germany, Italy, the UK, and the US with the clinical recommendations. Methods: DMD patients from Germany, Italy, the UK, and the US were identified through Translational Research in Europe – Assessment & Treatment of Neuromuscular Diseases (TREAT-NMD) registries and invited with a caregiver to complete a questionnaire with questions regarding DMD-related healthcare. Estimates of care were stratified by disease stage (early/late ambulatory/non-ambulatory) and compared against the care guidelines. Results: A total of 770 patients (173 German, 122 Italian, 191 UK, and 284 US) completed the questionnaire. Poor compliance to guidelines of routine follow-up by neuromuscular, cardiac, and respiratory specialists, physiotherapy, and access to medical devices and aids were observed in all countries. Less than 27% (209 of 770) of patients met all absolute recommendations, ranging from 9% (11 of 122) in Italy to 37% (70 of 191) in the UK, and from 49% (76 of 155) in the early ambulatory class to 16% (33 of 205) in the late non-ambulatory class. Conclusions: We show that the medical management of DMD varies substantially between Germany, Italy, the UK, and the US. Experience of real-world DMD care appears to be in poor agreement with the DMD clinical guidelines and increased compliance is urgently needed to improve treatment outcomes and enable patients to lead fulfilling, independent lives into adulthood.

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