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. 2018 Oct;142(Suppl 2):S43-S52.
doi: 10.1542/peds.2018-0333F.

Obesity and Endocrine Management of the Patient With Duchenne Muscular Dystrophy

Affiliations

Obesity and Endocrine Management of the Patient With Duchenne Muscular Dystrophy

David R Weber et al. Pediatrics. 2018 Oct.

Abstract

Duchenne muscular dystrophy (DMD) is associated with an increased risk of endocrine complications due to the effects of prolonged glucocorticoid therapy as well as progressive muscle weakness. Categories of complications include obesity and its comorbidities, short stature, pubertal delay, and adrenal insufficiency. Obesity prevention is important for long-term management of patients with DMD. Preventing glucocorticoid-induced weight gain fosters patient mobility, ease of transfer, and reduces sleep-disordered breathing. Metabolic complications from obesity (glucose intolerance, dyslipidemia) also can be avoided. Short stature and pubertal delay may negatively affect self-esteem and peer relationships, and careful monitoring of growth and pubertal development can allow anticipatory counseling. Adrenal insufficiency, a potentially life-threatening complication associated with prolonged glucocorticoid use, must be recognized so as to allow prompt treatment. In this article, we provide a summary of current guidance to ensure comprehensive endocrine management is followed in patients with DMD.

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Conflict of interest statement

POTENTIAL CONFLICT OF INTEREST: Dr Weber has previously served as a paid consultant for Marathon Pharmaceuticals; Drs Ward, Hadjiyannakis, McMillan, and Noritz have indicated they have no potential conflicts of interest to disclose.

Figures

FIGURE 1
FIGURE 1
Assessments and interventions for impaired growth and delayed puberty in patients with DMD. (Reproduced with permission from Birnkrant DJ, Bushby K, Bann CM, et al; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, an update, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17[3]:258.)

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