Steroids and Endocrine Issues mm

Steroids are proven to slow the progression of Duchenne and Becker, but they can cause a long list of worrying side effects, which makes the decision of if and when to start them a tough one. It is important that you discuss any concerns you have with your doctor before making the decision to start steroid therapy.

Steroids have been prescribed in Duchenne and Becker for over 20 years and more than 90 percent of boys in Australia now take them. The types of steroids used are ‘corticosteroids’ (also known as ‘glucocorticoids’). They are not the same as the anabolic steroids used by some body builders. It is thought that corticosteroids help in Duchenne and Becker due to their anti-inflammatory properties and by improving muscle regeneration.

Prednisolone and Deflazacort are the two types of corticosteroid prescribed for Duchenne and Becker. They each have pros and cons, which can be discussed with your doctor, but since prednisolone is much cheaper, this is usually tried first.

Steroids are usually started sometime between the ages of four and six, when motor skills have stopped improving, but have not yet started to decline. There is recent evidence that starting earlier is more effective, but this must be weighed against the risk of side effects.

Steroids are recommended in the international standards of care guidelines for Duchenne because of their ability to slow the progression of weakness, reduce the development of scoliosis (curvature of the spine) and delay breathing and heart problems. On average boys taking steroids are able to walk for three years longer. The long-term use of steroids can cause a number of possible side effects; the most common being weight gain, mood changes (irritability and hyperactivity) and around, puffy face. Some of the other possible side effects include difficulty sleeping, headaches, stomach irritation, growth suppression, delayed puberty, raised blood pressure, cataracts, bone fractures and increased susceptibility to infection.

After starting steroids your doctor will monitor the benefits and side effects. If the side effects are outweighing the benefit, the dose can be altered or tapered off completely. It is very important to not suddenly stop taking corticosteroids; their dose needs to be reduced slowly.

Checklist before starting steroids:

All routine immunisations should be given before starting corticosteroids

Make sure you know what to do if you forget a dose and where to get advice if needed. Be aware that a sudden stressful event such as illness, an accident or surgery, may require a higher dose of steroids for a short time. However, do not change the dose without discussing with your doctor.

Endocrinology & Bone Health 

There are many specialists that a person with Duchenne will need to see, and an Endocrinologist is an important member of the medical team. An Endocrinologist is a doctor who specialises in the endocrine or hormonal systems of the body. Long-term use of steroids can have an impact on the hormonal system of the body. This can affect growth and puberty, cause weight gain and adversely affect bone health.

An Endocrinologist specialises in a number of areas that they will assist with in Duchenne, these include, Steroids, Bone health and osteoporosis, Insulin resistance, Delayed growth, Hormones due to delayed puberty and weight gain.

Those with Duchenne commonly have weak bones, even before they commence steroid therapy, but this certainly worsens due to the side effects of steroids.

Endocrinologists are the experts to monitor steroids, the effects of the steroids and the management of side effects.

There are investigations they will request including:

Spinal X-rays – which are recommended yearly. This is to monitor how straight the spine is and any concerns with scoliosis, which is a curve in the back. If there are concerns X-rays will be requested every six months.

DEXA or DXA Scan (Dual – Energy X-ray Absorptiometry). This is an X-ray that measures the density of the bones and can help to diagnose thinning bones / osteoporosis. A base line DEXA is recommended after diagnosis and starts steroids. They will then be repeated every year.

Blood and urine tests – These tests are to monitor glucose, vitamin D and calcium levels. Also hormone tests from 13 years of age.

The Endocrinologist will also discuss vitamins and supplements, and work alongside the other specialists to coordinate the best care.