Duchenne muscular dystrophy (DMD) is the most common neuromuscular disorder in boys. It is linked to the genetically determined absence of the dystrophin, a major muscle fiber protein. DMD causes progressive motor deficit predominant in the root muscles and then spreads to the respiratory muscles and the heart, all together leading to premature death. With improved patient management (long-term corticosteroid therapy, ventilatory assistance), there is an increased longevity and, consequently, a higher frequency of cardiac complications.
In an article published in February 2020, North American specialists focused on the predictors of the age of death. For this purpose, the clinical and demographic data collected between 2005 and 2015 from 408 patients with DMD were analyzed. During this period, 29 deaths were recorded with a median age of 19 and a half. Almost a third of the deaths were of cardiac origin, and more particularly concerned patients who did not receive corticosteroid therapy. This observation could constitute an indirect argument for the prolongation of corticosteroids beyond the loss of gait. Furthermore, the finding of poor ventricular function figures before death was not necessarily predictive of death of cardiac origin.
Risk Factors for Cardiac and Non-cardiac Causes of Death in Males with Duchenne Muscular Dystrophy. Wittlieb-Weber CA, Knecht KR, Villa CR, Cunningham C, Conway J, Bock MJ, Gambetta KE, Lal AK, Schumacher KR, Law SP, Deshpande SR, West SC, Friedland-Little JM, Lytrivi ID, McCulloch MA14, Butts RJ, Weber DR, Johnson JN. Pediatr Cardiol. 2020 (Fév).