A new study has found that boys with Duchenne muscular dystrophy (DMD) on certain steroid regimes have a higher fracture risk than others.
The research was carried out by Dr Shuko Joseph and Dr Jarod Wong from the University of Glasgow in collaboration with researchers at the John Walton Muscular Dystrophy Research Centre, Newcastle. It was funded by Muscular Dystrophy UK, Action Duchenne and the Scottish Government Chief Scientist Office.
Using data from the UK NorthStar database, the researchers investigated growth and bone health of 381 boys on daily prednisolone, daily deflazacort, intermittent (ten days on, ten days off) prednisolone or intermittent deflazacort.
The results showed for the first time that boys treated with daily deflazacort had the highest frequency of fractures, including vertebral fractures (compression fractures of the back bone). The study also confirmed previous research that boys treated with daily deflazacort had the greatest degree of growth stunting, and that weight gain was greatest in those treated with daily prednisolone.
While these findings improve our understanding of the side effects of steroids, more research is needed to know which type of steroid and which regime is best. Steroids remain the recommended standard of care for DMD, and children receiving steroid treatment should be monitored in a specialist clinic. If you have any questions about your son’s treatment, please speak to your neuromuscular team.
Dr Kate Adcock, Director of Research and Innovation at Muscular Dystrophy UK, said:
We welcome these findings and hope that they will help pave the way to better care of people with Duchenne muscular dystrophy. However, there’s still a lot we don’t know about steroids and this study highlights the need for more research as well as regular monitoring of bone health.
The study was published in the medical journal, JAMA Neurology.
Dr Wong and the other researchers involved in the study have produced some helpful FAQs about steroids and DMD:
Steroids have been shown to improve muscle strength in boys with DMD. The exact mechanism is not clear but may relate to reduction in muscle inflammation. Studies have shown that steroids can prolong the age of loss of ambulation by about 1-2 years. They may also have protective effects on the heart and respiratory muscles. There is also evidence that steroids can reduce deformities of the spine (scoliosis). Steroids are the only proven therapy to improve the clinical outcome of boys with DMD.
There are currently two types of steroid medicines commonly used in DMD: Prednisolone and Deflazacort. They are usually given either daily or in an intermittent fashion: for example taking it 10 days on and then 10 days off, although in some countries they can be given in a different manner. It is very difficult to answer the question, as we do not have strong evidence of which steroid is better for improving muscle function. There is currently an international trial (FOR-DMD) ongoing that compares three steroid regimens, which will hopefully give us some answers.
Like any medicine, there are side effects. The side effects may vary from person to person. Weight gain including increasing roundness of the face (Cushingoid appearance), mood changes, and sleep disturbances are common. Thinning of the bones (osteoporosis) leading to fractures and stunted growth is also common. In adolescents, puberty may be late. Other possible side effects include cataracts, stomach irritation and thinning of the skin. It is therefore very important that a patient with DMD treated with steroids is monitored in a specialist clinic. It is very important that steroids are not stopped abruptly and that a plan should be in place for steroids to be given if a boy with DMD is very sick and cannot take steroids by mouth. Some side effects may be commoner with Prednisolone and some may be seen more frequently with Deflazacort. Sometimes, your doctor may discuss changing the type of steroid if there are a lot of side effects with the steroid that is being prescribed.
It is important that x-rays of the spine are done at least once every 2 years in a patient with DMD on steroids to detect vertebral fractures (compression fractures of the back bones). Blood tests to check vitamin D levels are also important. Please discuss this with your treating doctor and neuromuscular team.
We would encourage you to discuss this further with your treating doctor and neuromuscular team.
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Get in touch with the MDUK Research Line
Watch Dr Wong talk about bone health for Duchenne
We were able to fund this research thanks to the support of families and other donors who support our Duchenne Research Breakthrough Fund (DRBF). It is only through your contributions that we can continue to fund important studies like this. Donate now and help to change the lives of thousands of people living with muscle-wasting conditions. Thank you for your support.