What role do corticosteroids play in the management of Duchenne Muscular Dystrophy, and what are the potential side effects?

Role of corticosteroids in Duchenne Muscular Dystrophy (DMD): Corticosteroids, primarily prednisolone or deflazacort, are the mainstay of pharmacological treatment in DMD to slow the progression of muscle weakness and prolong ambulation. They improve muscle strength and function, delay the onset of scoliosis and respiratory decline, and may extend survival. Their use is supported by consensus clinical networks and guidelines recommending early initiation and long-term administration to optimise outcomes in DMD patients NICE NG243 Matthews et al. 2016Fontaine Carbonnel et al. 2024.

Potential side effects: Long-term corticosteroid therapy in DMD is associated with a range of adverse effects that require careful monitoring and management. Common side effects include weight gain, growth retardation, behavioural changes, hypertension, glucose intolerance, osteoporosis, and increased risk of infections. Specific concerns also include adrenal suppression necessitating adrenal insufficiency management protocols during stress or surgery NICE NG243 Czifrus & Berlau 2024Fontaine Carbonnel et al. 2024. Guidelines emphasise balancing the benefits of corticosteroids with these risks by optimising dosage and employing strategies to mitigate side effects, such as bone health monitoring and cardiovascular risk assessment NICE NG243.

In summary, corticosteroids play a critical role in modifying the natural history of DMD by preserving muscle function and delaying complications, but their use requires vigilant side effect management to ensure patient safety and quality of life NICE NG243 Matthews et al. 2016Czifrus & Berlau 2024Fontaine Carbonnel et al. 2024.