Replies to This Discussion

Permalink Reply by Sharyn Thompson on July 30, 2008 at 7:52am

Hi Jane,

James was diagnosed 2 years ago just after he turned 2. He was unable to walk up or down stairs, run, jump, ride a tricycle, and he kept falling down even when he was just walking. We put him on Deflazacort almost straight away, as we felt the need to "buy him some time" was more important than the side effects. Within a few months he wasn't falling as much (although still falls occasionally when he runs), running really fast, jumping continuously, jumping down from steps and landing on his feet, climbing up EVERYTHING, walking up and down stairs using alternate feet, swims like a fish unaided and getting his head up to breathe, and now he is able to ride a proper bicycle. He is still slower than other kids, but he does his damndest to keep up with them. I find he is getting tired a lot faster, but that is to be expected. With a little rest, he is back on his feet and active again.

The downside is probably the weight gain and the behaviour. He now weighs 22kg, but he doesn't look fat - he is just solid. Looking at the rest of my family and my husband's family, that could also be in his genes. My 3 nephews are exactly the same. His growth initially slowed down, but he has grown 5-7cms in the past 12 months and is now back on track.

His behaviour has become more agressive, but after talking to all the other mums at his preschool I have discovered their 4 year old boys are exactly the same. Having been brought up with girls, I have never been exposed to 4 year old boys and their "4-year-old shot of testosterone". He is a sensitive child, and this hasn't changed. His speech, cognitive and motor skills are fantastic after some work with specialists (and at home) and he is every bit a "normal little boy" who wants to be Superman!

Personally, I am glad we made the decision to put him on the deflazacort as the benefits have most certainly outweighed the side effects. However, maybe I need to revisit this topic in a few years and see if there are any more changes. HOpefully by then, PTC124 will be proven effective and he will be on that, and maybe won't need the steroids.

Something to consider, they say steroids provides a little more protection for the heart and cardiomyopathy is something practically all DMD boys end up with. This is certainly something to keep in mind.

My other advice is to make sure he is getting a good supply of vitamins and supplements. We give James a multivitamin with phytonutrients and probiotics, Omegas 3 + 6, antioxidants, CoQ10, Vitamin D, and Calcium. I don't know if they make MUCH difference, but I am sure every little bit helps and they won't be doing any harm.

Good luck,
Sharyn.

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Permalink Reply by Aimee Ealy on July 30, 2008 at 8:34am

The side effects that you are reading about are all possible side effects, not guarateed, and worse case senario. At his age, it important to start as soon as possible for the best benefit. Starting steriods is a big deal to every parent, but the benefits outweigh the negatives (in most cases). It is very scary, but in the long run it will keep him mobile longer, breathing better, and preserve his heart. As you have read about the bad things, read more about the positive studies, they outweight the negative. If the side effects are too much, you and your doctor can wean the steriods or switch to deflazacort or decrease the dosage. All the parents I have talked to either plan to start steriods soon or have started them. It is the best treatment we have now. Talk to a nutritionist before you start, and monitor him. If his appetite increases, increase his physical activity with swimming. Your son will not become a monster, but he may have some mood swings (PMS for boys?). Remember, it is considered the best treatment, and not irreversable. This is the first major decision you will have to make, and it is scary. But take the plunge, and think positive. It will not be as bad as you think. You survived the diagnosis, you can survive this!

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Permalink Reply by Brian Denger on July 30, 2008 at 9:39am

Hello Jane,


I have two sons (16 and 14) who have DMD and are still on steroids since diagnosis. We had concerns and read as much as was available ten years ago about the use and side effects for Prednisone. Our sons' doctor gave us the option while he leaned towards using Pred. Doctors are human and the desire to help is strong, so with little else available we can see why most advocate for steroids.

Even with steroids the course of muscle deterioration does not stop. Still, if there is additional strength and less fatigue my wife and I agree it has been worth the challenges. Our older son initially gained weight, so we chaanged from daily to twice a week dosing which helped. He stopped walking at age eight, but we continued Pred to maintian upper body function and support cardiac and respiratory function. Our younger son is fourteen and stopped walking independently last year, yet is still able to help us moving him on his feet very short distances. He is still able to throw a ball and had great upper body strength. Increased strength often offsets weight gain by additional caloric use.

We had no problems with behavioral issues. My understanding is steroids exacerbate moodiness. It may not be an issue for you as many families I know do not have to face this issue. The one thing to remember is this can be stopped or dosing altered if there are problems.

So, would I do it again if my sons were diagnosed today? Without hesitation. We are all "an experiment of one" meaning what works for one many not for another. With what is known about DMD the value to maintain ambulation is key. The longer boys walk the better for circulation, delay or prevention of scoliosis and it is better for their self esteem.

Side effects are still a concern, yet with careful diet and monitoring for gastric ulcers there is a place for Prednisone and Deflazacort.

Best of luck with your decision and with you son.

Brian Denger

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Permalink Reply by Kelvinsmom - Michelle on July 30, 2008 at 1:27pm

http://disability.ucdavis.edu/Patient%20Conf%202007%20DMD/DMD%20Tre...

First of all, I am sorry to hear of your son. My son too was diagnosed in January at age 5, only due to pushing and pushing for 4 years, yes I said, 4 years and then I wouldn't leave the doctor's office until I got blood test and xrays, etc.
The link above is nice to explain options that may or do help (a bit more scientific, but nice). There was a Deflazacort study presented I believe in Chicago (I can't find it) that is really great to show that the side effects are much less than Prednizone (less bone breakage, weight gain, etc.). It also shows, and doctors can tell you as such, that with steroids, there are very few kids requiring surgery for scoliosis, they walk many years longer on average, their heart and lungs fair much better, etc. I'm no doctor, but my son is on Deflazacort and is doing great. He started it over a month ago and he feels great and has tons of energy. So far, we haven't had any negative side effects, except he did pee his pants twice (which mind you, my son has always had trouble with this, as he waits until the last minute and has to run to the restroom). Of course, there can be many side effects as well, mood troubles, cataracts, weight gain, etc. The study did show that with Deflazacort, that boys on it weren't any more likely then kids who didn't take steroids to break bones. I hope I didn't confuse you, as I'm still in the "new" stage as well. Michelle

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Permalink Reply by Kelvinsmom - Michelle on July 30, 2008 at 1:38pm

http://www.parentprojectmd.org/site/DocServer/biggar_steroids_2006....

Here is one of the powerpoints that I used to determine giving it to Kelvin, along with his doctors. Michelle

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Permalink Reply by Sharyn Thompson on July 30, 2008 at 8:05pm

Gastric ulcers can also be avoided by taking the steroids on a full stomach. The best time to take them is immediately after breakfast. James always has his with yoghurt, as this way he can swallow 3 tablets without even knowing it.

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Permalink Reply by Sharon Good on July 31, 2008 at 7:07am

Steroids are both good and bad, as are all drugs. The best thing is to try, you can always stop. Liam was on pred from age 5 to 10. During this time we didn't have any problem with weight gain, but had some problems with the mood thing. When we started they were advocating a ten day on ten day off cycle to prevent side effects, however this resulted in a withdrawal period and an intial period that lasted up to 4 days each. As he was at the testosterone temperamental age anyway this was too hard, so we progressed to a one on one off cycle, which was fine.
Some kids have side effects some don't, you won't know until you try. We believe that the steroids kept Liam walking well until he got to big for his weakened muscles to cope. (at 10 he was only 10cm shorter than me!) We took Liam off steroids when we started to get "cushingoid" symptoms. Cushing's disease/syndrome is where the adrenal glands are not able to function properly, this results in kidney, liver, and thyroid problems, and a particular pattern of weight gain. Your Dr can explain this further.
My outlook has always been that we try what we can, we change what we can, so that both our sons and ourselves can live the best we can with this bugger of a disease!

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Permalink Reply by Kelvinsmom - Michelle on July 31, 2008 at 10:46pm

After a month on Deflazacort, Kelvin is running even better, he is pulling his knees up and is running pretty normal for the first time, my family couldn't believe the improvement seen, just today. He has always been able to run, but is doing better and better. I just hope his energy slows down a bit. It takes a bit to get him to sleep, he says he isn't sleepy, but I guess I'd rather have him feeling better than worry about my sleep. Hopefully when school starts, he will naturally be more tired. I'm also curious, has anyone on here had their child on Deflazacort or Prednizone and then found out that your child is BMD, not DMD? There has always been a question about Kelvin in this regard even though we are at DMD for now, so I just wondered. Also, would it matter, wouldn't you proceed the same no matter if your child had BMD or DMD? Michelle

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Permalink Reply by Jane Williams on July 31, 2008 at 10:51pm

The general concensus I'm getting is that Deflazacort is much better overall than Prednisone, we may try him on Pred for a few weeks and see how it goes,my other main concern is that Ryan also has Aspergers syndrome and I worry about how this affects him emotionally, I'm particularly worried about "Steroid rage".Thank you one and all for your feedback,much appreciated
Jane

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Permalink Reply by Karen Barnett on August 1, 2008 at 1:45am

Jane,
My son is more cognitively affected by DMD (has ODD, slow processing, sensory issues, etc) and I made the desicion to put him on steroids 2 yrs ago. They do say that boys that have more of the "cognitive problems" are more affected with the moodiness side effects and based on our experince I would most definatly agree with that. He has the roid rage and his behavior can be really bad on the steroids. I did notice that he was slower and "different" even before the diagnosis. I have read that the deletion area can affect boys more in the brain with the lack of dystrophin. My son is on deflazacort but a very low dose. He simply can't handle it with the higher recommended dose for his weight. I have tried countering the effects with mood stabilizers and have had some success with Lamictal. I really want to keep him on the steroids because they do make him stronger.

My advice to you is to try them and at least see what happens. They are the only thing that we have and they do increase strength & prolong function but you have to consider the quality of life if they affect your son's mood too much.. You can always stop them and the side effects are not lasting once they are out of their system. You can also try mood stabilizers like I have to acheive some kind of balance.

Hope this helps...

Karen

Jane Williams said:

The general concensus I'm getting is that Deflazacort is much better overall than Prednisone, we may try him on Pred for a few weeks and see how it goes,my other main concern is that Ryan also has Aspergers syndrome and I worry about how this affects him emotionally, I'm particularly worried about "Steroid rage".Thank you one and all for your feedback,much appreciated
Jane

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Permalink Reply by John Stewart on August 1, 2008 at 4:55am

Sharyn Thompson said:

Hi Jane,

James was diagnosed 2 years ago just after he turned 2. He was unable to walk up or down stairs, run, jump, ride a tricycle, and he kept falling down even when he was just walking. We put him on Deflazacort almost straight away, as we felt the need to "buy him some time" was more important than the side effects. Within a few months he wasn't falling as much (although still falls occasionally when he runs), running really fast, jumping continuously, jumping down from steps and landing on his feet, climbing up EVERYTHING, walking up and down stairs using alternate feet, swims like a fish unaided and getting his head up to breathe, and now he is able to ride a proper bicycle. He is still slower than other kids, but he does his damndest to keep up with them. I find he is getting tired a lot faster, but that is to be expected. With a little rest, he is back on his feet and active again.

The downside is probably the weight gain and the behaviour. He now weighs 22kg, but he doesn't look fat - he is just solid. Looking at the rest of my family and my husband's family, that could also be in his genes. My 3 nephews are exactly the same. His growth initially slowed down, but he has grown 5-7cms in the past 12 months and is now back on track.

His behaviour has become more agressive, but after talking to all the other mums at his preschool I have discovered their 4 year old boys are exactly the same. Having been brought up with girls, I have never been exposed to 4 year old boys and their "4-year-old shot of testosterone". He is a sensitive child, and this hasn't changed. His speech, cognitive and motor skills are fantastic after some work with specialists (and at home) and he is every bit a "normal little boy" who wants to be Superman!

Personally, I am glad we made the decision to put him on the deflazacort as the benefits have most certainly outweighed the side effects. However, maybe I need to revisit this topic in a few years and see if there are any more changes. HOpefully by then, PTC124 will be proven effective and he will be on that, and maybe won't need the steroids.

Something to consider, they say steroids provides a little more protection for the heart and cardiomyopathy is something practically all DMD boys end up with. This is certainly something to keep in mind.

My other advice is to make sure he is getting a good supply of vitamins and supplements. We give James a multivitamin with phytonutrients and probiotics, Omegas 3 + 6, antioxidants, CoQ10, Vitamin D, and Calcium. I don't know if they make MUCH difference, but I am sure every little bit helps and they won't be doing any harm.

Good luck,
Sharyn.

Hi Jane

As others mention there are quite a few issues with Steroids, but we are glad that our son Cameron has taken them for 5-years. He used Prednisolone for 4-years and Deflazacort for last 12-months. As you probably know Deflazacort is not readily available in Australia and Prednisolone is usually prescribed. I don't think there is a huge difference

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Permalink Reply by Sharyn Thompson on August 1, 2008 at 7:50am

Deflazacort is now available to the public in Australia apparently. James was on a trial through The Childrens Hospital at Westmead for two years, until we received notification in June that the trial had ended and the deflazacort was now available to all boys through prescription. Check with your doctors about this. Kristy Rose was James's trial coordinator - she is a member on here. You can probably ask her directly.

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