Glucocorticoid use can aid breathing ability in Duchenne adults: Study
Treatment also seen to maintain arm function in late stages of DMD
Glucocorticoid use can help maintain breathing ability and arm function in adults with Duchenne muscular dystrophy (DMD), a new study reports.
Importantly, the study found that glucocorticoid treatment was shown to be helpful even when only considering the time after patients had lost the ability to walk — which commonly occurs in the later stages of the neuromuscular disease.
“Our results provide evidence of the benefit of glucocorticoids in the late stages of DMD,” the researchers wrote, noting that glucocorticoid use was shown to “preserve late-stage functional abilities, [and] respiratory and cardiac function.”
“These findings have important implications for the care and equipment planning of adult clinical services,” the team wrote.
Their study, “Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids,” was published in the European Journal of Neurology.
Investigating glucocorticoid use when patients can no longer walk
Glucocorticoids, a type of corticosteroid, are medications that work to reduce inflammation by mimicking the activity of a hormone called cortisol. Routine glucocorticoid treatment is part of the standard care for DMD, and it’s well-established that glucocorticoid use in childhood can help slow disease progression, allowing patients to retain functional abilities like walking for a longer time.
There’s less data, however, on whether glucocorticoids have a meaningful benefit for older adolescents or adults with Duchenne who are no longer able to walk. That information, the researchers noted, could help in determining the “effective planning and provision of resources required for clinical services for adult individuals with DMD.”
To learn more, the team of U.K. scientists reviewed outcomes from 112 people with DMD who received care at their center between 1986 and 2022.
As of the most recent evaluation, the mean age of these patients was just older than 23, and the mean follow-up time was 18.5 years. The vast majority of these individuals were unable to walk by the time they were 18 years old, the team noted.
About two-thirds of the patients had started on glucocorticoid treatment as kids, while 25% had never received such therapy. Most patients who’d been on glucocorticoids as children continued taking these meds into adulthood, though the doses often were adjusted.
No standard guidelines exist for dosing glucocorticoids in adults with DMD, the researchers noted, highlighting a need for better standardization of care.
As of the most recent follow-up, slightly fewer than half of the patients were taking glucocorticoids.
Team says more research needed into treatment benefits for adults
Statistical tests showed that, compared with patients who’d never been on glucocorticoids, those taking the therapies as of the latest follow-up tended to have better scores in many measures of functionality. That included daily living activities such as getting in and out of a wheelchair, moving one’s arms and hands to eat or use a joystick, and coughing and swallowing.
Measures of breathing function and certain assessments of heart health also tended to be better in patients who’d continued on glucocorticoids through adulthood, compared with patients who’d never been on these medicines.
The main glucocorticoids used in this study were prednisone and Emflaza (deflazacort). Outcomes with either therapy were generally comparable, and further analyses only accounting for the time after patients lost the ability to walk yielded broadly similar results.
Safety data were in line with the known profile of glucocorticoid use. Common side effects included weight gain, weak, brittle bones (osteoporosis), and delays in the onset of puberty, all of which were reported in more than half of patients given glucocorticoids.
Overall, these data suggest that glucocorticoid treatment may be beneficial even in the late stages of DMD.
The researchers noted, however, that this analysis was limited by the small size of the sample. To that end, they concluded their study by calling for further research efforts “to confirm these findings by gathering comprehensive, agreed outcomes in a larger number of adults with DMD to inform patient care and management.”
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