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Northern Genetics Service

Steroids and Duchenne Muscular Dystrophy

This information has been produced to help you think about whether your child with Duchenne muscular dystrophy should have steroids or not. It includes some of the main questions people have asked in this situation and gives you some further information to look up. It is not intended to replace discussion with the muscle clinic team, so feel free to ask any questions you may have after reading this.

Why are steroids used in DMD?

It has been known for a number of years now that steroids have an effect on muscle strength in DMD. If they are used in boys who are still walking, they may have an effect on stabilising or even improving muscle strength for a period of time. Not all boys respond to steroids and the way that steroids have this effect on slowing the dystrophic process is not known.

What steroids are used?

The main steroid that is used is called Prednisolone (Prednisone in the USA). Deflazacort is also used in some countries and probably is just as effective. We are not talking about “anabolic steroids” which is what athletes use illegally to build up muscle – this does not have an effect in DMD.

What are the possible beneficial effects?

The studies which are now being reported on steroids are showing that overall boys who are treated with steroids walk for longer than those who are not. This effect varies from child to child but there are some studies coming out which are showing that some boys may carry on walking for years longer than they otherwise would. These children also seem to develop other complications of the condition less frequently too – so they develop breathing difficulties later and fewer problems with their spines.

There may also be a positive effect on the heart muscle.

What are the possible risks?

The down side of steroid treatment, and the reason that people are still very cautious about using them, is that they may have side effects. Steroids have many side effects, but the chances of getting these vary from person to person, and on the dosage and the regime used. There are also important things that we can do to try and protect against some of these side effects.

The most common side effects reported in the studies of using steroids in DMD in the short term are weight gain and mood changes. Weight gain seems to be most of a problem just at the time that the boys start taking the steroids, so it is a really good idea to keep a close eye on food intake at that time to avoid running into problems. We will be offering you the opportunity to speak to a dietician about this. In the longer term, after many years of treatment, there may be growth suppression, the development of cataracts and thinning of the bones. The risk of these long-term effects cannot be measured, but it is likely that the risk of different side effects varies with the different steroids used. Deflazacort on average may be associated with less weight gain, but a higher risk of development of cataracts (though these do not usually require any treatment).

Because children with DMD already have thin bones, it is very important to look after bone health, and the best way to do this is by promoting a healthy diet with good intake of calcium and vitamin D and lots of sunshine and exercise.

There are other possible side effects, which include raised blood pressure, diabetes, thinning of the skin and poor wound healing and increased susceptibility to infection, but these have not commonly been reported in the studies on DMD. A rare side effect of taking steroids in DMD is stomach irritation. It is important not to take non-steroidal anti-inflammatory tablets or medicines like Nurofen or aspirin while you are on steroids. If your son develops tummy pain, or there is any sign of bleeding, you should contact your GP. This may not mean that they have to stop taking the steroids but it is important that this is noted.

As the body gets used to having steroids, it is very important that they are not stopped suddenly.

How do the benefits and risks balance out?

This is a difficult question to answer because long-term controlled trials to look at this have not yet been done. The reason that it is important to consider using steroids is because the studies that are coming out now are showing some significant benefits. In some studies, some boys with DMD are still walking at the ages of 14 or 15.

However, there is no doubt that there can be important side effects, and the worst of these include significant growth delay and weak bones. It is important to try to minimise the risk of side effects by checking for them when you come to clinic. If significant side effects were picked up then the dose of the steroids might need to be altered or tapered off completely. It would also make sense to do this if it seemed like they were not having a positive effect. It is important to realise that if you do decide to use steroids this is not an “all or nothing” decision, but that the regime would be carefully worked out on an individual basis and changed if required.

What dose would be used and how often?

There are two main alternatives.

All of the studies that have shown a useful benefit of steroids have given them on a daily basis at a dose of 0.75mg/kg/day (the equivalent dose for deflazacort is 0.9mg/kg/day).

However, It is possible that using steroids intermittently (10 days on and 10 days off) could be effective while reducing the risk of side effects. It has not been proven in controlled trials though that this is as effective as using steroids every day.

At what age would it be considered to start and stop using steroids?

There is some experience now that suggests that starting steroids before boys start to lose significant muscle function leads to better results. All boys with DMD achieve a plateau of their motor function when they are no longer improving but not yet deteriorating. The age at which this occurs varies from child to child but is commonly around the age of 4-5 years.

Later on, when more function has been lost, there may still be benefits of starting steroids in improving strength and particularly the strength of the breathing muscles.

There is no evidence, as yet, for benefit of starting steroids in boys who have already lost ambulation, but studies looking at this are being done.

The age to stop steroids needs to be decided individually. It may be necessary to change or stop the steroids because of side effects. In other situations, they can be used for many years, beyond the age at loss of ambulation.

What happens if we decide to use steroids?

If you do decide that you want your son to use steroids, then there will be some extra checks that are necessary to make sure that they are having the desired effect and not causing any problems. It will be very important that you attend these appointments. Your son will need to have some blood and urine tests to check everything is OK to use the steroids, and to check that he is immune to chicken pox (if not he will need to be immunised against it as chicken pox can be very serious in children who are on steroids). He will also need a scan of his bones and an eye check (these will need to be repeated at intervals while he is on the steroids), and a good assessment of how he is getting on at the moment.

Once the steroids have been started, it will be important to see your son regularly to check for any side effects and see if there is any difference in your son’s physical performance and discuss with you if the dose seems right.

It is very important that steroids are not stopped suddenly but tapered off as the body becomes used to their effect and needs time to adjust if they are withdrawn. So if you decide you want to stop the steroids, that is fine, but he must do this under the guidance of a doctor. The dose has to be lowered gradually over several weeks to allow his own adrenal glands time to start making the steroids again themselves.

You need to get in touch with your GP or the muscle team if he is sick and can’t take his steroid for more than 24 hours, as he may need to have it by injection.

You will be given a steroid card to carry when your son gets his prescription. It might also be useful if your son wears a medi-alert bracelet or similar device when he is taking steroids. If he is sick or has an accident this will ensure that the doctor who is treating him knows about the treatment and that he might need to have steroids given to him.

What happens if we decide not to use steroids?

The follow-up of your son will carry on in the usual way and all other options will be discussed as they become relevant. If you change your mind and want to think about steroids again in the future, you just need to mention this at one of your routine appointments.

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