Glucocorticoid therapy is currently the most widely used treatment for Duchenne muscular dystrophy (DMD), having consistently shown to prolong ambulation by 2 years, reduce the frequency of scoliosis, and improve cardiorespiratory function. Among the most frequent side effects of glucocorticoids are fractures due to osteoporosis, linear growth retardation or arrest, and pubertal delay, the subjects of this review.
The diagnosis of osteoporosis has shifted in recent years away from a bone mineral density-centric to a fracture-focused approach, with particular emphasis on early vertebral fracture identification (one of the key triggers for osteoporosis intervention). Delayed puberty should be addressed in an age-appropriate manner, with numerous options available for sex steroid replacement. Growth impairment, however, is a more challenging complication of glucocorticoid-treated DMD, one that is most likely best addressed through growth-sparing therapies that target the dystrophinopathy.
With glucocorticoid prescription an increasingly prevalent component of DMD care, early attention to management of osteoporosis and delayed puberty are important components of multidisciplinary and anticipatory care. The treatment of short stature remains controversial, with no accepted therapy currently available to over-ride the toxic effects of glucocorticoids on the growth axis.
aDivision of Endocrinology and Metabolism, Children's Hospital of Eastern Ontario, Department of Pediatrics, University of Ottawa, Ottawa, Ontario, Canada
bDivision of Endocrinology and Diabetes, Golisano Children's Hospital, University of Rochester Medical Centre, Rochester, New York, USA
Correspondence to Dr Leanne M. Ward, MD, FRCPC, Professor of Pediatrics, Research Chair in Pediatric Bone Health, University of Ottawa, Ottawa, Ontario, Canada, Room 250H, 401 Smyth Road, Ottawa, Ontario, Canada K1H 8L1. Tel: +1 613 737 2253; fax: +1 613 738 4211; e-mail: Lward@cheo.on.ca