We have been presented with two different options by two equally passionate doctors. One doctor has prescribed weekend-dosing prednisone. She is basing the effectiveness of weekend dosing on published data that supports its effectiveness coupled with fewer side effects (versus daily dosing of either prednisone or deflazacort.) Another doctor prescribed daily deflazacort. She acknowledges the difficult side effect of diminished height but she believes that daily deflazacort will keep him walking for a longer period of time.
As parents of a five-year old, we are hoping that by the time he is ten, Henry will have other options. We want to hedge our bets, do the weekend prednisone and hope that in the next 5 years he will be able to have other medications that will keep him ambulatory. However, I notice that many folks on PPMD seem to prefer deflazacort.
What are your thoughts and why did you choose one course over the other?
Hi Claire, in 1997 with the data presented at that time, we started Anthony at 7-1/2 years of age on prednisone, 10 days on, 10 days off. The data presented at time from Douglas Biggar, MD from Canada seemed to indicate that Deflazacort had fewer side effects with behavior and weight gain. Since the FDA did not approve deflazacort, and doctors were still screaming at us for even thinking of putting our son on steroids anyway, we chose prednisone. We could not get a USA doctor to prescribe deflazacort let lone get it. The prednisone significantly increased the frequency and intensity of my son's negative behaviors. After three weeks, we had to stop. We then pursued getting Deflazacort in Canada and did so. Michael Brookes, MD was the doctor in Canada that saw Anthony. He prescribed a daily dose of Deflazacort. I do not recall the dose we started at, but he was on daily deflazacort (36mg) until 22. For us, we did not experience the behavior side effects like we did with Prednisone. His strenght and endurance immediately improved and then leveled off just as the data presented had predicted. Anthony did get some cataracts, but they we not an issue, and could be corrected if they became bothersome. His height did level off, so he was always short. I do believe this was a benefit to him as he was able to ambulate in walking KAFOs until age 21 with a weight of 85 pounds. We did get some osteoporosis, but that side effect was compensated for with Fosomax and weight bearing in KAFOs and Stander to maintain bone strength. For us, it was a good decision. Additionally, in the early days, we had to fly to Canada every three months to get Deflazacort refills and be seen by Doctor Brooke. Today, certain doctors in the USA will prescribe Deflazacort, and you can order the drug online from Masters. It is a tough decision to make. I mean, we as parents, with the best information available, have to determine how to proceed - what steroid, when to start, and what dosage schedule. It is not like taking your son to the doctor and being told he has strep and here’s the antibiotics. Yet, there is a lot more research out there to draw from, and more is still being done to get the best evidenced based protocol out there. I hope that helps.
We were on prednisone for a year, and our son's weight started to take off. Switched to deflazacort and the rate of weight gain slowed way down. Weight gain is the main reason to choose deflaz over prednisone - although everyone is different. Not everyone experiences weight gain on prednisone, and not everyone has less weight gain on deflaz. You'll pay out of pocket for deflaz, since it's not FDA approved. It's not reimbursable by insurance or a heath savings account. It's not terribly expensive, though. As Thomas mentions, you do need to get it overseas, but it's legal to import for personal use with a prescription and it's simple to do. Just requires a little advance planning, since you can't run to the drugstore to pick up a refill if you run out.
I don't think there's a strong concensus in the medical community as to dosing or schedule. There is concensus on the use of corticosteroids. They're part of the standards of care for DMD.
We began our son on Deflazacort almost 7 years ago at the age of 4. Today, at 10.5, he is very ambulatory, has no weight issues, behavioral issues or cushionoid face. In a recent Duchenne Connect webinar, information was released that evidence is trending to Deflazacort increasing ambulation to up to one year, but that could be affected by other factors.
The only time our son was on prednisone was for a bad case of poison ivy. The prednisone in that brief case, made him irratable and moody.
Our son does have growth delays caused by deflazacort, but that is no countered by Human Growth Hormone and he is growing at a rate of 3"/year. He is no longer the smallest boy in his class. :)
I would really recommend that you look at the Duchenne Connect webinar, that can be found on FB or the PPMD site. IT is very new information only, a week or two old, before you make your decision.
Good luck! It is a touch decision to make.
You can pretty much write our story the same as Lori's :) Our boys are the same age, started Deflazacort at the same time. They are the same size (we were together in January at Disney, both are thin. Both are on growth hormone. Lori's son is just more of a ladies man :)
Xavier has done so well on Deflazacort, I do not regret the choice for a minute. I have taken prednisone (usually about twice a year) when I get in a migraine crisis and I feel like crap! I admire how any kid can manage, they say the younger you are the better you can handle them. But I get evil, I am nauseated, I am AWAKE!!!! I have taken a deflazacort when I have a migraine once to see how it was and I really did not have the same issues as the predisone.
It is a choice you have to make for you and no choice is wrong. You just have to be confident in your choice for your child.
Thank you all so much for your input. The time you take to "talk" this out with me and share your stories helps so much. We are going to watch the webinar for additional information on deflazacort. In the meantime, Henry will start weekend prednisone this Friday. (I don't want to wait any longer to start steroids, even the little bit of time it might take to get deflaz. shipped.) So, as I told my husband, we better buckle our seatbelts.
Zachary is ten and has been on Pred since he was four. We have had absolutely no problems with behavior or other side effects of being on it. He has put on considerable weight in the past year or so, but I feel like that is not to do with the pred, but his breaking his leg in February 2011. Also, he is less ambulatory and that is the main problem with weight.
That said, make sure he is on calcium and vitamin D either way for bone health!
Probably a good idea to go ahead and start now and see how it goes. Good Luck!