TOPLINE

Boys with Duchenne muscular dystrophy (DMD) treated with daily corticosteroids maintained the ability to walk 4 years longer than those on intermittent treatment, but experienced significantly reduced height and higher rates of vertebral fractures.

METHODOLOGY

• Researchers conducted a retrospective cohort study to compare outcomes between daily deflazacort and intermittent (10 days on/10 days off) prednisone or deflazacort regimens in boys with DMD.
• A total of 219 patients were included from three neuromuscular centers in Belgium and the Netherlands, with 75 receiving daily treatment and 144 receiving intermittent treatment between 1995 and 2022.
• Median age at corticosteroid initiation was 5.6 years for daily treatment and 5.5 years for intermittent treatment, with mean follow-up of 10.7 years and 9.5 years, respectively.
• Outcome measures included motor function (loss of ambulation, upper and lower limb function), pulmonary function (forced vital capacity [FVC]), cardiac function, anthropometric measures (height, BMI), bone health (bone mineral density, vertebral fractures), and need for spinal surgery.
• Statistical models were used to compare outcomes between treatment groups over time.

TAKEAWAY

• Daily corticosteroid treatment resulted in later loss of ambulation compared with intermittent treatment (15.0 years vs 11.0 years, P < .001) and slower decline across measures of walking ability and upper limb function.
• Patients on daily treatment required significantly less spinal surgery for scoliosis (16.0% vs 27.1%, P < .05).
• Intermittent treatment was associated with greater final height at age 18 (170.2 cm vs 140.8 cm, P < .001), higher bone mineral density Z-scores (P < .001), and fewer vertebral fractures (P < .001).
• BMI across all ages, age at FVC < 60% of predicted, and annual decline in cardiac function showed no significant differences between treatment regimens.

IN PRACTICE

"These findings support informed, individualized treatment decisions and offer a framework for evaluating emerging therapies like vamorolone, which may provide comparable motor outcomes with fewer adverse effects," the authors wrote.

SOURCE

The study was led by Nadine A. Ikelaar, Leiden University Medical Center in Leiden, the Netherlands. It was published online on August 24 in the Journal of Neurology, Neurosurgery, and Psychiatry.

LIMITATIONS

The study drew patients from centers across Belgium and the Netherlands, where standards of care differed slightly between sites. Gaps in data from earlier years meant that age at loss of hand-to-mouth function and lung capacity decline could not always be fully captured. The analysis focused on a defined set of outcomes and did not capture several known steroid side effects.

DISCLOSURES

This study was funded by the Duchenne Parent Project. Two authors disclosed industry ties, including advisory board roles, research grants, and consultancies with multiple pharmaceutical companies.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.