Researchers found notable differences in the access to appropriate care for Duchenne’s muscular dystrophy (DMD) patients in seven European countries.
In a survey known as CARE-NMD, patients in DMD registries in the countries — Bulgaria, the Czech Republic, Denmark, Germany, Hungary, Poland and the United Kingdom — were questioned as to care practices and life quality. A total of 1,062 patients provided valid responses (861 children and 201 adults), and researchers analyzing them found significant inequalities between countries and age groups.
The average age of the participants was 13, and 53 percent reported not being able to walk. The median age at which participants lost walking ability was 10.3 years.
The study based on the results, “European Cross-Sectional Survey of Current Care Practices for Duchenne Muscular Dystrophy Reveals Regional and Age-Dependent Differences,” was published in November in the Journal of Neuromuscular Diseases.
Standard DMD care is recommended to include corticosteroids, physiotherapy, a regular echocardiogram and regular check-ups (once or twice per year) at specialized neuromuscular centers. Patients older than 6 are also advised to have their pulmonary function examined on a regular basis.
The survey demonstrated that ambulatory patients reported low adherence to these guidelines, and adherence among non-ambulatory patients was even lower. The most striking differences were visible mostly between Eastern and Western Europe.
“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. Extraordinary differences in access to physiotherapy were observed by country (for example, more than 90% in Germany, and less than 50% in the UK), and adults were less likely to receive physiotherapy than children,” Janbernd Kirschner, MD, the study lead investigator, with the medical center at the University of Freiburg, Germany, said in a press release.
Patient access to regular heart and lung assessments was also uneven, and nearly 25 percent of patients, particularly adults, did not get regular echocardiograms. Adherence to pulmonary function assessment guidelines was also low in patients who still hadn’t lost their ability to walk (62.8 percent), and very low among non-ambulatory patients (30.5 percent).
Differences between ambulatory and non-ambulatory patients were seen across most countries studied. For instance, in the U.K., nearly 66 percent of ambulatory patients received lung function assessment tests, but less than one-half of non-ambulatory patients were given that option. Patients also reported not being sufficiently informed about DMD-related breathing problems, and 31.3 percent of ambulatory — 39.9 of non-ambulatory — patients were not being aware of their current lung capacity.
In general, people visiting specialized neuromuscular centers at least once a year were found to receive more regular heart and lung checks, as well as corticosteroid medicines. They were also found to feel better informed about their illness and to be happier with their treatment. Receiving care from a neuromuscular specialist also decreased the duration of hospital stays after unplanned visits.
Better standardized care that meets international guidelines is essential to good patient care, the researchers said, and also important in the growing number of clinical trials underway in this disease.
“Standardized care allows researchers to be confident that any improvements seen in a trial are due to the drug being tested, rather than differences in care received by patients,” Kirschner said.
DMD affects 1 in 3,800 to 6,300 live male births and, while incurable, multidisciplinary treatment is increasing life expectancy well into adulthood.