I recently got a message on Facebook from the President of Parent Project Australia an organisation for the parents of children with Duchenne muscular dystrophy. This message started a conversation about various treatments for muscular dystrophy.

Duchenne muscular dystrophy is a genetic disease that mainly affects males. It is a degenerative muscle wasting disease. At birth a child with the defective gene appears to be perfectly normal. A child will slowly lose strength and they stop walking at age 9 to 12. At about age 15 weakening can begin to slow. In some cases the heart is affected and this can result in heart failure – sometimes suddenly. But if the patient is lucky their heart will remain relatively unaffected. Respiratory muscles can also fail, but there are treatments such as mechanical ventilation that stop this being a fatal symptom.

As many of my readers will know I have Duchenne muscular dystrophy I am now 29 and use a ventilator at night.

For this next bit to make sense you will need a bit of background information: In your lungs you have two different types of muscles: voluntary and involuntary. Voluntary muscles are used when holding your breath or when you take a deep breath. Involuntary muscles are the ones allow you to breathe without thinking about it, without these muscles you are unable to breathe when you are asleep. At around the age of 20 some boys with muscular dystrophy lose their involuntary lung muscles that allow them to breathe during sleep. Without mechanical ventilation the patient will die. This is exactly what happened to many of my friends.

You see it wasn’t until 10 years ago that ventilators were widely used to treat muscular dystrophy. It was commonly believed that anyone with muscular dystrophy would die before their 21st birthday. It can take a while before a change such as this is reflected in the medical textbooks. Duchenne can result in an early death, but this is not true in all cases. Some doctors continue to believe that Duchenne is a terminal illness.

I feel this belief is reflected in the type of treatments given to boys with muscular dystrophy. The focus of Parent Project Australia is in early diagnosis and early intervention. By intervention they mean physiotherapy and drugs. It is the drugs bit that I’m concerned about. Children as young as four are given steroids in an effort prolong the time they walk. I think parents and doctors are thinking about the short-term benefits and almost completely ignoring long-term ones. There are many studies that indicate children given steroids do walk longer, but, just look at the list of side effects: weight gain, hair growth, mood changes, stunted growth, decreased bone mass, acne and cataracts. I find this more than a little bit disturbing. There are no studies that I know of that steroids improve the long-term survival of someone with muscular dystrophy.

Just think what it would be like to be nine years old, you are progressively getting weaker and you know that you are going to be a wheelchair soon. This very stressful time often results in sometimes extreme behavioural problems – I know I’ve been through it. I can just imagine what taking a drug that has its own side effect of extreme behavioural problems would do. Early childhood is a very important time for someone’s emotional and psychological development. I feel this treatment could damage a child’s future prospects.

If you are a parent of a child with muscular dystrophy giving them steroids is not necessarily the best thing. You will make very hard choices had and you will make mistakes. It is important not to feel guilty about the decisions you have made or will make whether you choose steroids or not.

20 years ago when my brother and I made the transition from walking to wheelchair our family was given some valuable advice. Our parents were told not to put pressure on us to walk as this would just make it even harder for us and could even result in injury. We received our electric wheelchairs nearly a year before we actually stopped walking. This allowed us to adjust us to the idea of using electric wheelchairs and we learnt how to drive them before we needed to use them every day saving many plaster walls from certain doom. My brother and I both feel that this is the one piece of advice that made the most difference to us during this troubling time.

This entry was posted on Monday, February 25th, 2008 at 3:04 pm and is filed under I really don\'t know. You can follow any responses to this entry through the RSS 2.0 feed. You can leave a response, or trackback from your own site.