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. 2015;2(1):63-72.
doi: 10.3233/JND-140053.

Compliance to Care Guidelines for Duchenne Muscular Dystrophy

Erik Landfeldt  1   2 , Peter Lindgren  3 , Christopher F Bell  4 , Claude Schmitt  5 , Michela Guglieri  6 , Volker Straub  6 , Hanns Lochmüller #  6 , Katharine Bushby #  6
Affiliations

Compliance to Care Guidelines for Duchenne Muscular Dystrophy

Erik Landfeldt et al. J Neuromuscul Dis. 2015.

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.

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

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Figures

Fig. 1
Fig. 1
Visits to physicians and other healthcare professionals during the last six months. NEURO = Neuromuscular specialist. CARDIAC = Cardiac specialist. RESP = Respiratory specialist. ORTHO = Orthopaedist. PHYSIO = Physiotherapist. PSYCH = Therapist and/or psychologist. SLT = Speech and language therapist.
Fig. 2
Fig. 2
Current use of glucocorticoid medications (panel A) and regimens (panel B). Note: The figures above each column represent the proportion of patients in each stratum that were currently taking glucocorticoids. Alternate day: for example every other day. High-dose weekend: a high dose taken for example on Fridays and Saturdays. Intermittent: for example 10 days on, 10 days off.
See this image and copyright information in PMC

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