title: Compliance to Care Guidelines for Duchenne Muscular Dystrophy.
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.
authors: OptumInsight, Stockholm, Sweden.|Institute of Environmental Medicine, Karolinska Institutet, Stockholm, Sweden.
authors: Erik
authors: E
authors: Landfeldt
authors: Medical Management Centre, Department of Learning, Informatics, Management and Ethics, Karolinska Institutet, Stockholm, Sweden.
authors: Peter
authors: P
authors: Lindgren
authors: GlaxoSmithKline, Research Triangle Park, North Carolina, USA.
authors: Christopher F
authors: CF
authors: Bell
authors: GlaxoSmithKline, Brentford, Middlesex, UK.
authors: Claude
authors: C
authors: Schmitt
authors: Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK.
authors: Michela
authors: M
authors: Guglieri
authors: Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK.
authors: Volker
authors: V
authors: Straub
authors: Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK.
authors: Hanns
authors: H
authors: Lochmüller
authors: Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, UK.
authors: Katharine
authors: K
authors: Bushby
keywords: Muscular dystrophy; delivery of health care; duchenne; observational study; practice guideline