Steroids – counteracting side effects.

Steroids are a problem. Of course, they are Standard of Care and deliver benefit. There is no question about that. But over time, the side effects will hit you (your son) and it may be time to consider seeing an Endocrinologist. One of the members of the Cincinnati Neuromuscular Team is a pediatric endocrinologist. They evaluate every boy carefully and thoroughly . If he meets certain criteria, Growth Hormone is recommended. I spoke with 9 families whose sons are currently taking GH for more than 1 year. All are happy they initiated GH. They reported that the boys grew from 1-3 inches and felt that this growth positively impacted their quality of life. They were able to reach things previously out of their reach. Two were able to ride certain rides at Disneyworld. (that’s a BIG DEAL) . GH was insurance covered in all cases.

This does not mean GH is for every boy, rather it suggests this topic needs to be on the table. The Cincinnati team will soon have date on 30+ boys. Physicians who care for boys with Duchenne and physicians outside the field who care for children are chronic steroids (pediatric rheumatologists, ped. Pulmonologists, etc) need to collaborate and discuss ideas, risks and benefit.
Bone health is also an important topic in this area. There is considerable interest in the effect of low level whole body vibration for short periods of time each day. In post menopausal women and the elderly, this type of vibration has been demonstrated to improve bone density. Why not Duchenne?

And puberty. One parent called to say their son’s friends had difficulty assisting with the urinal. Because this young man had not gone through puberty, it was difficult to use the urinal from his chair and typically resulted in wet underwear. An endocrinologist recommended testosterone and it helped! No more problems and other benefits as well. But again, this treatment will not necessarily work for all of our boys and as always we must remain cautiously optimistic.

As more results develop, we will share what we learn with you. Also, you may be interested to know that we are developing an international meeting to discuss these issues. More on that in the coming weeks.

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Comment by Pat Furlong on January 27, 2009 at 1:24pm
Hello Julie, Sounds like you made the right decision for Brandon. I'm really happy you are not seeing any side effects. I realize he has just been on only three weeks, but wondering if you have noticed any changes in strength. It may still be a bit early (sometimes 6 wks), but I thought I would ask.
Comment by Julie Hathaway on January 27, 2009 at 1:16pm
My son, Brandon, began using Deflazacort on January 6, 2009. He is 6 years old and was diagnosed 3 months ago, today. We haven't seen any side effects or benefits at this point. We purchased it from Canada through a website. Our neurologist recommended it and so did Dr. Wong in Cincinnati. We prayed about it and feel that it is the best option for Brandon. Good luck and God bless you.
Comment by Michelle Scaglione on January 23, 2009 at 7:44pm
My son is on deflazacort. You can email Dr. Biggar or Dr Wong to get their opinion on what to do about the steroid once the boys are no longer ambulatory. I chose to keep my son on it after his lost the ability to walk.
Comment by Holly Cahoon on January 23, 2009 at 6:17pm
What steroid are your boys taking? My son was taking Prednisolone, He actually had a doctor appointment today and we have the option of putting him back on, at a lower dose, I'm just concerned about the side effects. I've heard good things about Deflazacort, although I don't believe it's available in the United States, does any one know about this steroid, or which ones would you recommend?

Comment by Pat Furlong on January 22, 2009 at 9:55am
Also Holly, have you (or your doctor) considered a reduced dose. It might have the effect of preserving arm strength and cardio protection without significant gain. Just a thought.
Comment by Holly Cahoon on January 21, 2009 at 7:27pm
Yes I have noticed his arm muscles are getting a lot weaker. I'll have to talk to my doctor about the ace inhibitor for his heart. Thanks for the suggestion.
Comment by Michelle Scaglione on January 21, 2009 at 6:05pm
My son is 16. I still have him on steroids because I was told it will help slow down the decline of his arm strength and keep his spine straight as long as possible. Were you given the option to keep him on steroids? I understand your point about weight gain and moon face. Also I have my son on an ace inhibitor to help his heart. His heart is fine at this time. The ace inhibitor is used so his heart doesnt have to work as hard.
Comment by Holly Cahoon on January 21, 2009 at 1:12pm
My son was on steroids from the time of his diagnosis, at age 3 up until about 6 months ago. My son is now 12. The steroids definitely helped him, he was stronger, walked much longer than other boys his age with Duchenne, I would say the biggest side effects with my son was his mood swings and weight gane. Once my son was ambulatory at age 11 we kept him on the steroids for the reason of continuing to help make his heart strong, etc. Our doctor however had us take him off the steroids due to the suvere weight gane, his doctor believed the weight gane would be harder on his heart than the steroids doing good for his heart, since he was already full time in his wheelchair we decided to stop the steroids. His moods swings got much better and he is down to the regular weight a 12 year old should be. I think other children looked at him different in his wheelchair because of the weight and how big and round his face got. He looks total normal now and is happier. I would definitely reccomment the steroids however in the beginnning for your sons, his quality of life was better taking the steroids to keep him walking as long as he did.

Comment by Pat Furlong on January 19, 2009 at 3:32pm
Janine, Drs. Wong and Rutter make the decision to use (or not) GH based on a certain number of criteria. We do not have evidence to suggest that there is a higher percentage of boys with Duchenne who are deficient in growth hormone. We do know of less than 5 boys (guessing on the number) who have both DMD and GH deficiency.
It is the chronic use of steroids that causes significant growth delay.
Here is the issue. Steroids are now considered Gold Standard in Duchenne, for all the right reasons - improved strength, extended ambulation, benefits to respiratory and cardiac. Boys are now taking steroids from early on (diagnosis ~4-5years) for more than 10 years. And while there is benefit, there are concerns on the other end of the spectrum. Teenagers do not want to be heads shorter than their peers. They have expressed concern about delayed puberty, about facial changes due to steroids and other issues related to quality of life. Often parents 'judge' benefit looking at only one outcome -walking. The boys have a different view of quality and as adolescents/teenagers are now expressing their own concerns. I do not think the issue is simple, nor do I think there is a 'one size fits all' about endocrine issues. I do think it is an important issue to put on the table, seek the best advice from physicians who use chronic steroids in other indications and develop a strategy for moving forward.

Comment by Pat Furlong on January 19, 2009 at 3:17pm
Non-ambulatory boys are often continued on steroids based on the effect on upper body strength, respiratory and cardiac. It is the extended walking that maintains the integrity of the spine and prevents scoliosis. Dr. Biggar's data suggests that most, but not all boys who take steroids are able to avoid scoliosis surgery.

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