Pharmacological therapy: Learn more about treatment with glucocorticoids

Along with physiotherapy, treatment with glucocorticoids remains a mainstay of Duchenne muscular dystrophy treatment and it is recommended that individuals should continue taking glucocorticoids after loss of ambulation.1

Recent studies have also demonstrated the benefits of early glucocorticoid treatment, before significant physical decline.1-3

The long-term effects of glucocorticoid therapy have been shown to include*:

muscle

Preserved muscle strength and motor function4,5

stairs

Delayed loss of ambulation1,4

lungs

Preserved pulmonary function1,4,5

spine

Avoidance of scoliosis surgery1,4,5

Improved survival4

*compared to untreated patients

The 2018 International Care Guidelines recommends following an initial consultation with the family, a discussion regarding side effects and a nutritional consultation should occur before any glucocorticoid treatment is initiated.1 Well documented side effects of long-term corticosteroid use can include; weight gain and obesity, acne and warts, cushingoid features, growth retardation and delayed puberty, cataracts, immune/adrenal suppression, glucose intolerance, hypertension, adverse behavioural changes, gastro-oesophageal reflux, peptic ulcer, gastritis, osteoporosis and myoglobinuria.6

Click here for the International Duchenne Care Considerations summaries.

1. Birnkrant DJ, et al. Lancet Neurol. 2018;17:251–267 [Part 1].
2. Merlini L, et al. Muscle Nerve. 2012;45:796–802.
3. Lamb MM, et al. J Pediatr. 2016;173:207–213.e3.
4. Gloss D, et al. Neurology. 2016;86:465–472.
5. Bushby K, et al. Lancet Neurol. 2010;9:77–93.
6. Nascimento Osorio A, et al. Neurologia. 2019;34(7):469-481.

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