Improved Outcomes in Duchenne Muscular Dystrophy With Long-Term Glucocorticoid Treatment – Neurology Advisor

Lifelong use of glucocorticoids is associated with physical strength and function improvements, greater health-related quality of life, and improved survival in patients with Duchenne muscular dystrophy, according to findings from a prospective cohort study published in the Lancet.

Investigators enrolled 440 male patients with Duchenne muscular dystrophy from a total of 20 health centers during 2006-2009 and 2012-2016. Treatment with glucocorticoids for <1 month was compared with glucocorticoid treatment of ≥1 year, with researchers evaluating the differences between the 2 groups in regard to progression of clinically meaningful mobility and upper limb milestones.

Among participants managed with glucocorticoids for ≥1 year, time to all disease progression milestones was longer than for those treated <1 month or those never treated with the therapy (P <.0001).

Compared with treatment <1 month, patients managed with glucocorticoids for ≥1 year were older at loss of upper limb milestones by 2.8 to 8.0 years and at loss of mobility milestones by 2.1 to 4.4 years. In addition, the use of deflazacort was associated with a higher median age at loss of 3 milestones by 2.1 to 2.7 years when compared with treatment with prednisolone or prednisone (P <.012).

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