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.