Adult care for Duchenne muscular dystrophy in the UK

Survival in Duchenne muscular dystrophy (DMD) has risen in recent years due to iterative improvements in care. This paper describe the results of the CARE-NMD survey of care practices for adults with DMD in the UK in light of international consensus care guidelines. We also compare the UK experience of adult care with the care available to pediatric patients and adults in other European countries (Germany, Denmark, Bulgaria, Czech Republic, Hungary, and Poland). UK adults don’t experience as much comprehensive care as children in their access to specialized clinics, frequency of cardiac and respiratory assessments, and access to professional physiotherapy. Access to the latter is especially poor when compared to other European adult cohorts. While the total number of nights in hospital (planned and unplanned admissions) is lower among UK adults than elsewhere in Western Europe, social inclusion lags behind other Western European countries. We observe that attendance at specialized clinic is associated with more frequent cardiac and respiratory assessments among adults, in line with international best practice. Attendance at such clinics in the UK, though comparable to other countries, is still far from universal.

With a growing adult population living with DMD, and cardiac and respiratory failure the leading causes of death in this population, it is suggested that there is a need for an urgent improvement in adult access to specialized clinics and to consistent, comprehensive best practice care.