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Daily corticosteroids help boys with Duchenne muscular dystrophy walk 4 years longer than intermittent regimens, but the trade-off is significant: stunted growth and more vertebral fractures. A retrospective study of 219 patients from Belgium and the Netherlands found daily deflazacort delayed loss of ambulation to age 15 vs. age 11 for intermittent treatment. The findings push clinicians toward more individualized treatment decisions.
Boys with Duchenne muscular dystrophy (DMD) face a tough trade-off when it comes to corticosteroid treatment. A new retrospective study published in the Journal of Neurology, Neurosurgery, and Psychiatry found that daily corticosteroids kept boys walking nearly 4 years longer than intermittent regimens — but came with meaningful costs to bone health and physical growth.
The study followed 219 boys across three neuromuscular centers in Belgium and the Netherlands between 1995 and 2022. Those on daily deflazacort lost the ability to walk at a median age of 15, compared to age 11 for those on intermittent prednisone or deflazacort. Daily treatment also reduced the need for scoliosis surgery. However, boys on intermittent regimens were significantly taller at age 18 and had better bone density with fewer vertebral fractures.
By the Numbers:
Why it matters: These findings give clinicians a clearer picture of the real-world trade-offs between DMD treatment regimens, and set a benchmark for evaluating newer therapies like vamorolone, which may offer similar motor benefits with fewer side effects.
