Today’s Solutions: June 26, 2022

Many of us listen with apprehension when a doctor suggests steroids because they have a bad reputation, and justifiably so. Steroid use can have long-term consequences like stunted growth, reduced bone density, and even compromised glandular function. 

As patients, we should get all the information we can before using doctor-recommended steroids, so it’s encouraging that new research shows that steroid therapy can help children with Duchene muscular dystrophy (DMD).

DMD is a condition that almost exclusively affects boys and appears around age three or four. It is characterized by great muscular weakness, and left untreated it can lead to severe physical disability, even eventually spreading to the heart and breathing muscles, making the disease fatal. In the US, over 28,000 people have DMD. 

“Corticosteroids are likely to remain the main treatment for DMD worldwide for the foreseeable future, so it is critical that we establish a standard of care that is backed by scientific evidence,” said neurologist Robert Griggs MD.  “This study shows that health concerns over the daily use of corticosteroids are overstated and that there is a clear benefit in terms of improved motor and pulmonary functions. These findings clearly support the daily regimen over an intermittent one as an initial treatment for boys with DMD.”

Corticosteroids prednisone and deflazacort are proven treatments for improving muscle strength in DMD patients and are a commonly used regimen. The mention of steroids still worries patients and parents, though, and many doctors implement ten-days-off-ten-days-on regimens to limit children’s chance of side effects such as weight gain, stunted growth, and loss of bone density.  

An international study led by Dr. Robert Griggs examined 196 boys with DMD at 32 research centers throughout Europe and North America over three years. The participants were divided into three groups with daily regimens of deflazacort or prednisone, or intermittent prednisone. The finding showed that daily regimens outperformed the intermittent group in improved strength and muscle function with minimal serious side effects. 

While there were still side effects worth considering for treatment, these findings reinforce support for these kinds of steroids being used to treat muscular dystrophy. 

Source Study: University of Rochester Medical Center NewsroomDaily Steroids Safe and Slow Progression of Duchenne Muscular Dystrophy | URMC Newsroom (rochester.edu)

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