Analysis Reveals Wide Global Disparities in Prevalence, Burden, Cost and Treatment of DMD

Analysis Reveals Wide Global Disparities in Prevalence, Burden, Cost and Treatment of DMD

The journal Orphanet Journal of Rare Diseases has published a review highlighting the prevalence of Duchenne muscular dystrophy (DMD) as well as the disease’s costs and treatment patterns in North America and Europe.

 

The analysis, “The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review,” retrieved 9,850 titles from searches, and narrowed that down to 58 studies published between 2005 and 2015. These covered such topics as birth and mortality, point prevalence (number of individuals with the disease at any given time), severity and progression, and quality of life.

The analysis found that DMD’s prevalence ranges from 15.9 to 19.5 per 100,000 live births. But that varies widely; in the United States, 1.9 out of 100,000 have the disease, compared to 2.2 per 100,000 in Great Britain, 6.1 per 100,000 in Canada, and 10.9 per 100,000 in France.

In a center in France, the median survival for adult DMD patients born between 1970 and 1994 was 40.95 years, whereas those born between 1955 and 1969 had a median survival of 25.77 years.

“There are important evidence gaps, particularly in measuring prevalence and mortality, although people seem to be living longer with the condition, which may be partially as a result of more widespread prescribing of corticosteroids, improved access to ventilation and development and publication of more specific and thorough care guidelines,” researchers wrote.

The analysis also revealed that DMD boys lose the ability to walk at a median age of 12, and ventilation starts at about 20 years.

“Disease severity in the prevalent DMD population also appears to be high, in that at any given time and any given country between 22 and 56 percent are likely to have lost ambulation and between 27 and 57 percent have cardiomyopathy [heart disease],” they added.

The study also found large variations among countries regarding prescriptions of corticosteroids, scoliosis surgery, ventilation and physiotherapy. In Germany, advanced DMD costs about six times as much to manage as earlier stages of the disease.

“Indirect costs are a significant feature of this condition and should have a role in informing appropriate care packaging and coordinated financial planning of health and social care,” researchers wrote. “Per-capita cost burden increases with disease progression.”

Leave a Comment

Your email address will not be published. Required fields are marked *