Corticosteroid Treatment in Duchenne Muscular Dystrophy

Who is this leaflet for?

This leaflet is for parents and caregivers. It explains how certain medications - called corticosteroids - can help manage Duchenne Muscular Dystrophy (DMD).

What is Duchenne Muscular Dystrophy?

DMD is a genetic condition that causes progressive muscle weakness in children - typically boys. Over time it affects their ability to walk and can impact the heart and lungs.

What is the purpose of corticosteroids in the treatment of DMD?

Corticosteroids (sometimes just called “steroids”) help reduce inflammation and slow the progression of muscle damage in children with DMD. The use of corticosteroids aims to maintain muscle strength for as long as possible.

What treatments are currently available?

There are three different corticosteroid medications available for treating children with DMD. You and your doctor will choose one.

Currently there is no clear evidence that one corticosteroid is better than another, overall. Each works to reduce inflammation and help preserve muscle strength.

There is no right or wrong choice, it depends on your child and what matters most to your family.

The three available corticosteroids are:

• Prednisolone
• Deflazacort
• Vamorolone

Prednisolone and deflazacort are usually given on a “ten days on/ten days off” pattern. This means you take the medication every day for ten days then have a ten-day break before starting again. Vamorolone is usually given every day.

Sometimes other dosing patterns, such as every-day dosing or weekend-only dosing, are recommended. You and your doctor can discuss whether this might be suitable for your child.

When should treatment start?

Treatment with a corticosteroid should usually start by the time your child is four years old, or sometimes earlier. If your child is diagnosed after the age of four, treatment will be started soon after diagnosis.

Vamorolone cannot be started before the age of four years. Deflazacort and prednisolone can be started at any age.

Can children switch treatments?

Yes. Some children may switch between corticosteroids if they experience too many side-effects or if their function declines. You can switch as many times as necessary.

If you switch to vamorolone from either of the other two steroids, you may need extra monitoring in case of a complication called “adrenal insufficiency”. Sometimes it may be necessary to take an additional steroid medication temporarily to manage this.

What are the benefits?

Corticosteroids are not a cure for DMD, but they can provide important benefits:

• slowing down muscle weakening
• helping preserve walking ability and everyday function
• delaying heart and breathing complications

What are the side-effects?

Unfortunately, all corticosteroids can cause side-effects. These may be different for different people but they can include:

• weight gain due to increased appetite
• behavioural changes
• high blood pressure
• high blood sugar
• immune suppression (meaning you are more vulnerable to infections)
• reduced bone strength leading to a risk of fractures
• reduced growth
• risk of cataracts (an eye problem)
• skin changes
• adrenal insufficiency: this is when the body stops producing its own natural corticosteroids, so if you stop taking corticosteroid medication suddenly you can get dangerously ill

When your child starts taking corticosteroids, you will be given some more detailed information about steroid safety and monitoring. You will also be given a blue ‘Steroid card’, which will highlight that your child is taking a corticosteroid and information about how to minimise risks when taking a corticosteroid.

If your child develops chicken pox, or comes into contact with someone with chicken pox or shingles, it is very important that you contact your doctor straight away, as a medicine might need to be prescribed to protect your child.

Is there a “best” treatment?

There is no good evidence that any of the three corticosteroids is more effective at preserving muscle strength than the others. However, it is possible that vamorolone may be slightly less effective.

All three corticosteroids can cause any of the side-effects above. However, the balance of one side-effect with another may be different:

• Prednisolone may cause more weight gain but less growth stunting.
• Deflazacort may cause less weight gain but more growth stunting.
• Vamorolone may cause less growth stunting compared to the others but might be slightly less effective in preserving muscle strength.

The different corticosteroids and patterns of dosing which we use have mostly not been tested directly against each other. There has been a study called FOR-DMD which looked at the options for optimal dosing regimes for steroids. Your neuromuscular team can discuss this with you, if needed.

Alternatives

Corticosteroids are recommended for most children with DMD, including those taking other medication such as ataluren or givinostat. Whether or not a child is taking corticosteroids, supportive therapies such as physiotherapy, heart and lung monitoring and vitamins for bone health are also important.

Medication

When you come to the clinic or if your child is admitted to hospital, please bring all their medicines (including inhalers, injections, creams, eye drops or patches) and a current repeat prescription from your GP. Please tell the ward staff about all the medicines your child uses. During your child’s stay, if you wish to administer your child’s medication yourself, please speak to their nurse. Pharmacists visit the wards regularly and can help with any medicine queries.

MyChart

We would encourage you to sign up for MyChart. This is the electronic patient portal at Cambridge University Hospitals that enables patients to securely access parts of their health record held within the hospital’s electronic patient record system (Epic). It is available via your home computer or mobile device.

More information is available on the My Chart section on our website.

Contacts/further information

Neuromuscular nurse specialists/ Secretary:

Tel: 01223 348882/ 01223 274250

E-mail the CUH Paed Neuro Muscular Nurses