Glucocorticoid-Associated Demise of a Patient With Duchenne Muscular Dystrophy

Abstract

We describe the clinical deterioration of a 26-yr-old man with Duchenne muscular dystrophy on oral daily high-dose deflazacort. Although this daily regimen was targeted to benefit ambulation and respiration, it resulted in premature death with lethal sequelae from liver failure, decubiti, diabetes mellitus, and morbid obesity. This case illustrates the need for further research weighing risk versus benefit of daily glucocorticoid therapy, specifically deflazacort, in Duchenne muscular dystrophy patients. Thus, curtailment of daily dosing to eliminate dire sequelae in patients living longer into adulthood than ever before is recommended.

Metabolic assessment in children with neuromuscular disorders shows risk of liver enlargement, steatosis and fibrosis

“…Glucocorticoid‐induced metabolic disturbances are risk factors for NAFLD 26 . A recent case report described a 26‐year‐old patient with DMD who developed lethal steatosis and liver failure after 10 years of glucocorticoid treatment 27 . If the accumulative doses of glucocorticoids are the only explanation for the liver enlargement, steatosis and fibrosis in the DMD patients, we would expect an increase in the number of patients with DMD with liver involvement with increasing age.…”