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Duchenne Muscular Dystrophy
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Long-term treatment can present significant challenges
by
Paul Smyth, MD
August 1, 2024
Patients with Duchenne muscular dystrophy (DMD) are prescribed corticosteroid anti-inflammatory drugs as standard of care, but long-term use comes with significant adverse effects.
For individuals with DMD, corticosteroids have been the only treatment shown to significantly impact physical function, observed Tina Duong, MPT, PhD, director of neuromuscular medicine clinical outcomes research at Stanford University in Palo Alto, California.
“Boys undergoing glucocorticoid treatment often experience improvements in their ability to run, walk, and perform daily activities,” Duong told MedPage Today. “Over the long term, these patients also exhibit a slower disease progression compared with those not receiving glucocorticoids.”
The side effects of glucocorticoids present significant challenges for families and caregivers, Duong pointed out.
“Weight gain, Cushingoid appearance, behavioral difficulties, and stunted growth are some of the potential adverse effects that require careful consideration,” she noted. “This necessitates a close collaboration between families and their clinical teams to weigh the benefits of glucocorticoids against these possible side effects.”
In addition to prednisone and prednisolone, both used off-label, two corticosteroid formulations have been approved for DMD in recent years: the glucocorticoid deflazacort (Emflaza) in 2017, and the dissociative steroid vamorolone (Agamree) in 2023.
Deflazacort
Deflazacort is approved for patients ages 2 years and older; a generic version for patients ages 5 and older is now available.
In 2016, a phase III trial of prednisone versus deflazacort showed that both agents improved muscle strength compared with placebo, but deflazacort was associated with less weight gain.
The deflazacort label carries warnings about endocrine, immunologic, cardiovascular, renal, gastrointestinal, behavioral, bone, eye, skin, and vaccination concerns. The most common adverse events with treatment are Cushingoid appearance, increased appetite, weight gain, upper respiratory infection, cough, pollakiuria, hirsutism, central obesity, and nasopharyngitis.
A prospective cohort study in The Lancet that evaluated the long-term effects of glucocorticoids in DMD, including deflazacort, found that treatment was associated with reduced risk in loss of mobility and upper limb function milestones. Subgroup analysis found deflazacort was associated with an increased median age at loss of three clinical milestones by 2.1 to 2.7 years compared with prednisone or prednisolone (P
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