Gloucocorticods, a form of steroid hormone medication, are among the standard prescribing practice for patients with Duchenne muscular dystrophy (DMD) in many clinics. They aren’t, however, the therapy of choice for all doctors.
Without long-term data proving the drug’s effectiveness, some doctors have delayed treatment with glucocorticoids because of the significant side effects that their use can cause, like weight gain, delayed growth, and immunosuppression. Consequently, many parents have been hesitant in allowing their children to be treated in this way.
A new study published online in The Lancet
could convince these decision makers of the long-term benefit, though, as it supports previous data.
DMD, a progressive neuromuscular disease, most commonly affects males and is characterized by progressive muscle weakness and an eventual loss of muscle function. It is the result of low levels of the dystrophin protein, which is intended to work as a shock-absorber for muscles. Without it, the affected muscles gradually die. Glucocorticoids are often administered to DMD patients as the condition progresses, and studies over the past 30 years have confirmed only the short-term benefit that they can provide.
DMD typically begins to present in the lower bodies of patients 4- or 5-years old, and those with the condition are likely to be non-ambulatory by their teenage years.
"Everyone had the idea that long-term use could be beneficial, but nobody had really rigorously tested that," said Heather Gordish-Dressman, Ph.D., a statistician at the Center for Genetic Medicine Research at Children's National Health System and study senior author in a press release
In the study, 440 male patients with DMD aged 2-28 years were enrolled during 2 recruitment periods and followed for up to 10 years. The researchers compared treatment without glucocorticoids to a cumulative treatment of 1 year or longer. Nine disease-related and clinically meaningful milestones, most of which pertained to mobility and function of the upper limbs, were used to measure the effects of the drug. Going from supine to standing, climbing stairs, and walking or running 10 meters were among the tests used in the evaluation process.
The study confirmed prolonged preservation of muscle strength and function, as well as a decreased risk of death. Time to all disease progression milestone events was meaningfully longer in patients treated with glucocorticoids for 1 year or longer when compared to patients treated for less than 1 month or never treated.
"This long-term, follow-up study provides the most definitive evidence that the benefits of glucocorticoid steroid therapy in DMD extend over the entire lifespan. Most importantly, patients with Duchenne using glucocorticoids experienced an overall reduction in risk of death by more than 50 percent,” said Craig McDonald, M.D., a University of California, Davis, professor and lead author of the study.
Emflaza (deflazacort), the first corticosteroid approved by the U.S. Food and Drug Administration (FDA) for the treatment of DMD, was the primary drug used in the study, and is currently available in both tablets and oral suspension.
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- McDonald CM, Henricson, EK, Abresch RT, Duong T, Joyce NC, Clemens PR. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study. The Lancet. 2017;390(10110):2325-2412. doi: 10.1016/S0140-6736(17)32160-8.
- Glucocorticoids offer long-term benefits for patients with Duchenne muscular dystrophy [news release]. Washington: Children’s National Health System; November 27, 2017. eurekalert.org/pub_releases/2017-11/cnhs-gol112117.php. Accessed November 27, 2017.