I need some opinions about stopping deflazacort. I know each kid is different and of course most of us are not docs, but any advice would be appreciated.
My son is almost 7 and has been taked deflazacort for 3 years. He taked a high dose 2xwk- to help to minimize his behaviors during the school week. This means he is extra emotional and aggressive during the weekend.
We were originally told he has becker's, but this is not the case. My sons walking is poor- he can only walk on flat surfaces, not the slightest incline or decline or he will fall. He does not walk outside except on a driveway and he cannot walk more than 20 feet or step over anything. he crawls in the house, but has a very very difficult time getting into any chair- he must pull up on furniture and he struggle so much. he typically asks to be helped up off the floor. he uses a stroller or his powerchair when out of the home. he does walk around a little in his classroom and he has a rifton chair too.
18 months ago we did a trial off of the deflazacort and his energy level decreased and we out him back on this medication- however he was also more ambulatory at that time.
I feel like he only continues to decline- no improvement, like other stories I read about. I am happy that his energy level seems increased, but it does not really improve his ambulation. With the scary possible side effects (he had catarat extraction this summer, I was told they came on very suddently and did not look like medication enduced cataracts), only decline, and aggressive and emotional behaviors I am really thinking it is time to accept that this medication is just not a great option for our situation.
Any advice? did anyone have a child with a similar story or a quick progression with ambulation?
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Thanks Fred...I'm looking for a family therapist in our area to see if an "outside" person is easier for Lucas to talk to. Being 14 isn't easy no matter what...DMD/steroids/ ect greatly aggravate it. I wish Lucas could let go like your son does....he's more the hold everything inside until he can't take it anymore and thus the meltdowns. He has also been participating in the GSK exon skipping trials that just got put on hold. He feels very betrayed by the drug companies, doctors, ect. because he could really see a difference while on the meds. Another concern of mine is how those drugs mix with the deflaz as far as emotional issues go. Thanks for your concern......Will contact you if I have questions!
Fred Luchetti said:
We are both members of Facebook's Drisapersen Trial Participants. If you would like to discuss the trial and/or deflazacort/behaviour issues of 13/14 year old boys with DMD, please message me on either site.
Karen flor said:
Fred Luchetti said:
Keep your child walking for as long as you can. Kyle broke his ankle at the age of 13 and never walked again. I have never heard of giving weekly doses of a steroid because of the fat soluble nature of the steroid. Giving a high dose weekly might taper off the level of the drug to the point of being non effective at the end of the weekly cycle.This is also why there is no difference if you take them in the morning or spread the dose out during the day. The steroid gets into your system and works throughout the day and if there is any tapering off, it happens while the child is sleeping.
Fred I'm sure there are different reasons why some boys are on prednisone vs. deflazacort. One of the likely reasons is that in the US it is not FDA approved and there for not covered by insurance. It is quite an expense to pay for it outright. I am sure that some families simply can't afford it. My son took prednisone for many years before switching to deflazacort. We switched to it after he was in a chair full time (at around 15) because as I'm sure you know it has less incidence of weight gain. We researched and made the best decision for our son with the information we had gathered. Isn't that what we all do? I am happy you are confident in your decisions regarding your son and his medications. I am as well
It is sad the way the Medical Industry in the US rips you guys off at every turn. I pay about $150 for three months of Deflazacort which is 80% refunded by my wife's insurance. I am not in any way criticizing your choices but rather the options that you have available to you. The US is the last place you want to be with a chronically ill family member.
Fred, you are quite lucky that your wife's insurance will cover the deflazacort at 80%, as no one else I have spoken to about this gets any coverage for it, since it is not approved here in Canada either, just available through Special Access. I do however use it as a medical expense on our income tax, so I feel it all works out in the end anyway. I just paid $54.80 yesterday for a 2 month supply (of course it is being subsidized at some level, we are not paying the actual price), so I am not complaining about the cost, but certainly would understand that in the U.S. some folks just cannot afford it at the non-subsidized price.
Even here in Montreal one of the pediatric hospitals doesn't even tell the parents about deflazacort, just automatically prescribes prednisone, which I find totally unfair not to inform them that an alternative exists. I have been told by the other children's hospital (the one where my son is followed and where I work in the lab) that does recommend and prescribe deflazacort that it is because the other place just doesn't want to go through the hassle of filling out special access forms every 6-months (not sure if that is the real reason, but it certainly seems likely). Yes, sad as you say.
I was able to get Deflazacort covered under our provincial healthcare for boys with DMD. It took quite a while to do it, but all we pay now is shipping since the only pharmacy in Saskatchewan is about 300KM away from us.
Ideally, we wouldn't have to have our kids on steroids at all, and we are all very aware of the side effects that prednisone and deflazacort can cause. However, these side effects are different for every child. Just because one child doesn't have all the side effects from one of these drugs, doesn't mean the next one won't.
The Care Considerations document suggests that for most of our children, prednisone and its side effects are very tolerable. Here's some quotes from that document regarding switching between the two:
Deﬂazacort might be preferred to prednisone for some patients because of the likely lower risk of weight gain.
If obesity is of concern, then the physician should consider switching treatment from prednisone to deﬂazacort
Consider switching from deﬂazacort to prednisone if cataracts evolve that aﬀect vision
Frankly I would choose weight gain and longer life than what is offered elsewhere. I would like it understood and I will state it once again. I am NOT trying to scare or criticize the parents of DMD kids. They are not experts in the field and are trusting the words of the practitioners in their area. However... these practitioners know full well the benefits and risks of both drugs and every medical practitioner who I have spoken with on the subject would prescribe Deflazacort over Prednisone just to avoid the damage that long term Prednisone use does that long term Deflazacort use does not. So my criticism is of the medical community as a whole that keeps peddling this drug when a better choice is available
There is weight gain with Deflazacort too. My son could lose a few pounds and he knows it. He also has cataracts which only affect his vision when looking through telescopes or microscopes. He also has never taken any drugs to protect his heart or his liver from Deflazacort. To suggest that these two drugs are on an equal footing is a very dangerous position to be taking, they are not equal and as I have stated before, IN MY OPINION, Deflazacort is the better alternative.
Finally, yes, I would love for my son to be off steroids. Since he is 19 now if he had never taken steroids my May 24 routine every year would be to visit his grave as he would have long passed. The steroid is a necessary evil and one that I embrace as I do my son every single day.
Children will react differently to these steroids (delfazacorte and prednisolone) and what might work for some doesnt nescessarily mean that it will work for others.
Recently we decided to try switching from prednisolone to deflazacorte and it was the worst thing we could have done. My son is now 8 and almost a year ago he rolled his ankle which broke his tibia and fibia clean across. He wasnt able to have it pinned either as the break was too close to his growth plates and so he was externally cast. After he had the cast off and was walking again, we upped his roids due to his increase in weight and found his behaviour to be intolerable. So we switched to deflazacorte. His behaviour improved out of sight, however he slowly lost all strength to the point that he was struggling to get around the house and found it difficult to get up stairs (before he was going up them like you and I)
We lasted about 6 weeks before we made the decision to switch back to prednisolone. His behaviour has become really hard to deal with again, but he is back to the strength levels he was before.
Everyone has to make the decision about what is best for their son.