Guidelines for the use of growth hormone in children with short stature.

Drug and Therapeutics Committee of the Lawson Wilkins Pediatric Endocrine Society
Mary M. Lee, University of Massachusetts Medical School

At the time of publication, Mary Lee was not yet affiliated with the University of Massachusetts Medical School.


On the basis of the information currently available, the only conditions in which GH therapy appears to be safe and effective in increasing adult height are GH deficiency and, likely, Turner syndrome. Therapy with GH also increases the growth velocity of children with CRI and may increase adult height, but no long-term data are available. Encouraging short-term results have been reported in patients with a few other conditions, such as patients with glucocorticoid-induced growth failure, renal transplantation, and Prader-Willi syndrome, but the data are limited and no long-term studies have been reported; in many other conditions the data are either inconclusive or discouraging. For children in these latter groups, GH therapy should be considered investigational and undertaken only as part of ethically sound, controlled clinical trials. Knowledge concerning the conditions in which GH is safe and effective is a prerequisite to making rational decisions concerning its use. However, in deciding whether therapy is warranted in an individual child, one must consider other important factors. The age and emotional maturity of the child, the family structure and dynamics, and even financial considerations may, in some cases, outweigh the presence of a GH-responsive condition. Likewise, the child's and the family's views about "short" stature and the likely benefits of therapy must be considered. Ultimately, a decision concerning the appropriateness of GH therapy must be individualized and based on a realistic assessment of its impact on the quality of life of the child and future adult.