Depressed left ventricular contractile reserve diagnosed by dobutamine stress echocardiography in a patient with Duchenne muscular dystrophy.

Cardiomyopathy is a leading cause of death in patients with Duchenne muscular dystrophy. Congestive heart failure is often sub-clinical and unrecognized as a result of the severe physical limitations of this patient population. We report the case of a 16-year-old boy with Duchenne muscular dystrophy who demonstrated normal left ventricular systolic function at rest by screening transthoracic echocardiogram. This patient, however, was noted to have depressed left ventricular contractile reserve by dobutamine stress echocardiography. Dobutamine stress echocardiography can have an important role in unmasking subclinical heart failure in this patient population.

Corticosteroids in Duchenne muscular dystrophy: a reappraisal.

Duchenne muscular dystrophy is the most common and most severe form of childhood muscular dystrophies, resulting in early loss of ambulation between the ages of 7 and 13 years and death in the teens and twenties. Despite the phenomenal advances made in the understanding of the molecular genetics of the disease, no definitive cure has been found. Of all of the therapeutic drugs studied in Duchenne muscular dystrophy, only prednisone seems to have the potential for providing interim functional improvement for boys with Duchenne muscular dystrophy while they wait for a cure with gene or cell therapy. There is still no consensus regarding recommending corticosteroids as standard therapy for boys. This is an evidence-based review of all of the studies of corticosteroids (prednisone, deflazacort, and oxandrolone) in Duchenne muscular dystrophy. From this review, it is clear that until a definitive treatment for Duchenne muscular dystrophy is available, the use of deflazacort and prednisone with judicious dietary control and close clinical monitoring for side effects seems the best intervention for interim preservation of function in such a common devastating disorder of young growing boys.