Hi Carol,

I am sorry to hear that the doctor is too busy to meet and can't understand why your daughter would have any questions!!!  He is suggesting shots for your grandson everyday for (possibly) the next 5-8 years; and at a (possible) total cost of well over $100,000---for something that may or may not work--- and he doesn't think there is anything to talk about! At the very least, one would think that he would be willing to discuss what hope there is for good results!!  Perhaps, it's because he has no idea.  Please forgive the pop psychology, but I have found that the best doctors are usually the ones who are more than happy to answer questions.  The ones who are defensive and evasive are often that way to cover up their own insecurities and inadequacies. 

I will be the first to admit that I am no doctor, but having been down this road with my son, do have our experience to share.  Along the way, I have also collected a number of different abstracts and studies on growth hormone treatment, and would be happy to share them with your daughter, if she is interested. 

There are quite a few studies on Growth Hormone treatment out there, but very few specifically on those with CAH.  What the studies generally show is that growth hormones are most effective on children who have a growth hormone deficiency.  In cases of short children without growth hormone deficiency (what would be termed "idiopathic short stature") results are usually no more than 1-2."  Now, I am not at all knocking one or two inches.  When every inch counts, an inch or two is nothing to thumb one's nose at. But, it is only fair that your doctor tell you this ahead of time, so you understand what the situation is, especially if you will not be part of a study protocol, in which the cost of the medication is borne by a grant or some other type of funding.   

When evaluating results, I think it is important to look at statistics showing final, rather than interim, heights attained.  Growth hormones do seem to increase height rates ( known as growth "velocity") over the short term (in the first 2-3 years, or so) but it still remains to be seen whether or not final height is significantly greater, when all is said and done.  In other words, they can make a child grow faster, but not necessarily change what they would have achieved, to begin with, over a longer period of time. 

Because the short stature associated with CAH does not have to do with growth hormone deficiency, those with CAH might be expected to respond to treatment similarly to those with idiopathic short stature.  As I said, most studies on children with idiopathic short stature, seem to show only modest gains, though one study conducted by Genentech---one of the 3 companies that manufactures growth hormone---shows significantly better results.  The reasons why are unclear. 

There is only one study on growth hormone treatment in CAH children, that I am aware of, and that is the one being conducted at Cornell Medical Center in NYC, which has also been expanded to include some of the affiliate hospitals in the NYC area.  They have just published a paper (in 2001), showing results achieved after two years of therapy, in a group of 20 CAH patients.  The data looks promising, though, again, the numbers are based on interim, rather than final, heights.  This was the study that my son was in. 

When we first enrolled, I had expected that there would be reams of data from many patients who had undergone the therapy, and was quite surprised to be told that final height numbers were available for only ONE patient, after 16 years.  With nothing to lose, but possibly something to gain, we nevertheless decided that it was worth a try.  You may or may not feel the same way, since you are in a slightly different situation. 

At any rate, I don't believe the doctor should be pressuring your daughter into something she is reluctant about, but instead, should be helping her objectively weigh the pros and cons.  It is natural to have questions when faced with a decision such as this---who wouldn't, when faced with a long-term commitment, such as this?  To me, the fact that he is unwilling to entertain them speaks volumes, in and of itself. 

If you are interested in these studies and would like to email me directly, you can do so, by clicking on my name in blue.