When to start steroids... not really the same question as in other posts though

I have mixed feelings about when to start. Our boy is 4, diagnosed at 2. The doctors say he is doing so well that they want to hold off on starting the steroids. While genetics put him in this mess, other genetics are helping him because he is very flexible and has a lot of muscle elasticity. They believe this is why his symptom progression is very slow. At this point, he holds on to railings for stability to climb up and down stairs. He has a small waddling gait with running. He gets a small amount of air under his feet with jumping. My only physical concern is his resistance to learning how to write since his finger grasp is weak.

One mom mentioned to me that starting him on steroids now would allow him to keep and improve on what he already has for physical abilities. She suggested that if I wait until he loses some of his physical capabilities, then the steroid may not be as helpful for trying to recover some of those physical abilities. FYI, I still need to run this by the docs too.

I was wondering if anyone has any has seen the case of not recovering some of the physical losses when starting steroids after you notice an increase in DMD symptoms. Does anyone else agree that starting steroids while things are going better than average for his age is a good idea? Or is a good idea to wait until we start noticing degrading abilities?

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My son Nicolas is 7 and diagnosed at 4. He started steroids (Deflazacort), just after his 5th birthday. I wish we would have started sooner, wish he would have been diagnosed sooner. One thing is that I believe it does help with stabilization. The other point I wanted to make is that Nicolas was pretty okay at 2 and at 4. He just got a little more tired and had a gait, didn't run very fast either. The progression happens no matter what, and now that Nicolas is 7 - I see him having more and more difficulties. The Cinderella period is over.

By the way, not only is steroids supposed to stabilize, but it's also helpful for the lungs and heart in the future. I'm not a fan of prednisone and have read the side effects in detail for both drugs. Do some homework and make a decision that is comfortable for you and your family.
Dear Friends,
Care Considerations (cdc effort to develop standards of care) will be published in Lancet, January and February issue. Care considerations guidelines for steroids suggest that a steroid regimen should be implemented when the child reaches a plateau. This is clearly a conservative approach (difficult to get 15 or so experts to agree) as some of the more aggressive physicians recommend starting early, as early as 3+ years of age. It is certainly not an easy discussion. Here's what we do know. Steroids (for most, but not all boys) improve function. They also push some of the behavior 'buttons', in that behaviors are exaggerated. Again, not all boys. And there are physicians who suggest the earlier you interveine, the less impact on behavior. Makes it clear as mud right?
So, here are my thoughts and feel free to throw them out the window if you disagree.
1. I think starting early is good. Boys improve between the ages of 3-7 years without any intervention (called golden years) but steroids certainly ramp up the effct. Many of the boys are able to run fairly well, jump (both feet off the ground), ride bikes. This improves self-esteem and this is essential for success.
2. Early, like 3 or 4. Keep in mind, you do not have to start off with daily if you are worried about how this will affect him as well as the impact on growth. You might consider testing the water with a weekend regimen or low dose (ask your doc). Give yourself (and your son) 6 weeks. You will be gain some insight into effect on strength, behavior, weight gain,etc.
3. If he has a good response, behaviors are managable and he is not eating you out of house and home (or you are working through the diet/nutrition issues), you will be able to increase the dose as you/your doctor see fit.
As a mom, I would find steroids at 3 yrs really scary. Taking it easy, understanding the effect, collecting information will provide a basis for next steps.
And it is important to preserve and protect muscle function early on. The field is moving fast (well, fast for scientific research, not for parents who need answers yesterday). Boys are expected to live long lives and become adults. They will need self-esteem! And to the degree possible, protection of muscle function and access to therapies. We are well aware, building/improving function is possible. Restoring function that is lost is very difficult at the moment.
So, these little guys have a distinct advantage. As parents, jumping into the deep end is frightening, but sticking a leg in to test the water will help you build your knowledge base and your confidence for next steps.
Warm regards - please do not hesitate to email me directly or call: pat@parentprojectmd.org; 513-424-0696.
Always, always ask your doctor, but keep in mind, you/your son have the highest stakes in this.
pat
If your deletion is high on the list of potentials for exon skipping, consider that once the muscle becomes fibrotic, it cannot be restored by exon skipping. If you think that will become a therapy for you at some point, you'd want to protect as much muscle as possible...
This is a discussion I am very interested in. I know all about the benefits and the side effects. These are my questions:

1. Is there any scientific evidence that starting steroids at age 3 is more beneficial than starting at 5? Any published papers, studies or unpublished data from clinicians?
2. Are there any other neuro teams besides the one at Cincinnati Children’s who recommend starting at such an early age (3-4)? I asked a few clinicians in other teams but they do not recommend starting so early due to the side effects.
3. How is the self esteem of the 8-9 year old boys looking like 5 year olds b/c of the stunted growth after being on steroids for 5-6 years? I am quite concerned about that age when they really start understanding what DMD entails. The decision would be easier knowing that their self esteem is still good at that age.

Thanks.
Ofelia, I can't speak to #1 or #2, but on #3 - my son is 10 and is about 10%tile for height. He's never once mentioned anything about his height, so I don't think it's a factor in his self-esteem at all. That's him, though, other kids, I'm sure, are different.
I like these perspectives and questions. It's hard as parents to help our children to preserve muscle function when they want to go run and play, climb and jump because those are things we all love to do as kids. So there is this fine line to balance that preservation of muscle with preservation of childhood.

Early on in the diagnosis, I was set on not doing the steroids. However, I found research that showed how well the drug promoted better spine health which of course meant better respiratory function. If anyone can help post links her that may answer some of the questions Ofelia asked, this may help.

Pat, as always your perspectives provide relief. I forgot there are options to test the drug out "toe first" rather than diving in blindly. Thank you chiming in here.

Liisa
Keith, my oldest boy who does not have DMD is under the 10% tile in growth. He hasn't mentioned any issues with friends at school about his height either =) Liisa

Keith Van Houten said:
Ofelia, I can't speak to #1 or #2, but on #3 - my son is 10 and is about 10%tile for height. He's never once mentioned anything about his height, so I don't think it's a factor in his self-esteem at all. That's him, though, other kids, I'm sure, are different.
ofelia,

Our son dylan is now 8. His self esteem is very much intact. Despite being much shorter than others in his class, he has never mentioned that as an issue. He has mentioned other issues like being slower and falling more. He started steroids at 3.5yrs. He is never mistaken for a five yr old, maybe late 6 or 7? Big maturity difference between 5 and 8. My understanding is that height dies become important to many of the boys, but not at this age as much as early jr high etc and at that point they do look much younger.

carrie
This helps. Thank you Keith, Liisa and Carrie.
Ofelia,

My son is 8 and was diagnosed at 4 ( almost 5 ). He's been on steroids since just after his 5th birthday. Like Carrie said, he could be mistaken for a 6 or 7 year old, but I don't think 5. He's in second grade now and though he's definitely one of the shortest in his grade, at this point there are several other kids who are simply short and about the same height as him. Though I know this will change each year as his classmates grow and he doesn't, right now at 8 it's not as obvious. As far as his self-esteem I don't think it's had much of an impact yet. He did just ask me the other day why he had turned 8 and not gotten any taller, but my 4yr. old has been on a kick where he's always talking about being tall like dad when he grows up. I think that's what prompted him to ask. I do know the day is coming when his brother ( who's tall for his age ) will pass him up, and I worry about how it will make him feel to have his brother who's four years younger be bigger than him.

Kim
Thanks Kim.
Thankfully my son is 2 and I have some more time, but it is quite scary to think at him at 3 taking steroids. Can they even swallow those pills at 3? Are the pills small enough?

One more question. Is there any scientific evidence that starting the steroids early (around age 3) at lower doses (as Pat suggested) or alternate day or weekend dose provides more benefit than starting them at the regular daily dose at age 4-5? Any articles or studies? It is very difficult for me to make a decision based on one doctor’s opinion and I cannot find any other doctor recommending them so early. If the benefits of starting them earlier outweigh the side effects why isn’t this recommended by more doctors? What is the practice outside US in Canada or Australia? My understanding is that Muntoni’s team at GOSH in UK is not recommending them at such an early age. Very difficult decision!
Though we go to cincinnati now, we got the original recommendation to start at 3.5 from docs/researchers at Childrens National Medical Center in DC 5 yrs ago. After we started Dylan, we went to Kevin Flanigan for are for the the next two years (Salt lake city, UTAH) and he certainly didnt object, seemed to think it was a good/reasonable choice, though we had already made it. I dont think there have been any real studies reporting on starting so early, which is why the recommendation is 5. They dont have data arguing against earlier, I think they just dont have data on any earlier. Perheps there will be some retrospective data by the time your son is 3. At the time we stared , it was based on dr anecdotal evidence/opinion . There were not that many starting early then. I dont think anyone really knows about the long term consequences of starting at 3 vs 5. We started Dylan on prednisone and then switched to deflazacort a couple years later. The prednisone came in a liquid form that we gave him. Generic tasted awful but for a little more money one could get flavored stuff my son liked and willingly drank. He learned to take pills at 5 ish and we switched him to pred pills and then deflaz pills. Dont know if there is a liquid deflaz formulation.

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