Significant differences in care found for patients with Duchenne muscular dystrophy

Significant regional and age-dependent differences in access to care were seen for Europeans with Duchenne muscular dystrophy, according to research published in the Journal of Neuromuscular Diseases.

In the CARE-NMD survey, a European project to improve care for Duchenne muscular dystrophy (DMD), researchers examined care practices and quality of life through DMD patient registries in Bulgaria, the Czech Republic, Denmark, Germany, Hungary, Poland and the United Kingdom.

Overall, 1,062 responses were received from 861 children and 201 adults. The average respondent’s age was 13 years and 53% lost the ability to walk at age 10.3 years.

“Access to corticosteroid treatment, which has been shown to slow the progression of DMD, varied between countries and by age: only 21% of those who had lost the ability to walk remained on corticosteroids,” Janbernd Kirschner, MD, lead investigator in the Department of Neuropediatrics and Muscle Disorders, Medical Center at the University of Freiburg, Germany, said in a press release. “Extraordinary differences in access to physiotherapy were observed by country (for example, more than 90% in Germany and less than 50% in the [United Kingdom] U.K.), and adults were less likely to receive physiotherapy than children.”

According to the release, the surveys showed adherence to guidelines was low in ambulatory patients and lower in non-ambulatory patients, with significant differences between Eastern and Western European countries, as well as within Western Europe.

Access to regular heart and lung function tests also varied, the release noted. Adherence to pulmonary function assessment guidelines was 62.8% in patients who were ambulatory and 30.5% among patients who were non-ambulatory. It also varied notably between countries for both groups. In addition, many patients reported being insufficiently informed about DMD-related breathing problems, with 31.3% of ambulatory and 39.9% of non-ambulatory patients knowing their current lung capacity.

According to the release, researchers also found patients who visited specialized neuromuscular centers at least once per year tended to receive more frequent heart and lung checks and corticosteroid medication, were better informed about their condition and were more satisfied with their treatment. Care from a neuromuscular specialist also reduced the duration of stay during unplanned admissions to hospitals, the release noted.

Kirschner and coinvestigators suggested that access to synchronized, best-practice care along with international recommendations is associated with improved life expectancy and quality of life for patients with DMD, and added it is also important to the increasing number of clinical trials currently underway testing treatments with a more curative approach.


Kirschner J, et al. J Neuromuscul Dis. 2016;doi:10.3233/JND-160185.

Disclosure: The researchers report no relevant financial disclosures.

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