American Association of Clinical Endocrinologists Position Statement Growth Hormone Usage in Short Children

The AACE Medical Guidelines for Clinical Practice for Growth Hormone Use in Adults and Children do not currently address the issue of the use of growth hormone in short children. The following provides AACE's position on this new indication.

In August 2003, the Food and Drug Administration (FDA) approved the use of Humatrope, a biosynthetic hormone, to treat children who are unusually short. The FDA based its decision on studies that found Humatrope added between one and three inches in height to children who took it for four to six years, and that there were no significant short term health risks to the children.

Biosynthetic growth hormone has been available since the late 1980s, but had been approved by the FDA only for shortness caused by specific diseases or syndromes. The new indication is for children who are short without a known cause, (i.e., < - 2.25 standard deviations below the mean and have an adult height prediction of less than 5'3" for boys and less than 4'11" for girls).

The American Association of Clinical Endocrinologists (AACE) has always maintained that responsible use of growth hormone (GH) means consulting a physician, who can determine whether such therapy is indicated by appropriate diagnostic testing and proof of GH deficiency in children with open epiphyses. With the FDA's recent approval of a new indication for GH use in children without GH deficiency, AACE continues to recommend responsible use of GH. First, AACE advocates that growth hormone should be administered and monitored by pediatric or adult endocrinologists with training and expertise in this area. AACE also believes that currently there is limited information on long-term effectiveness and safety of this treatment in short children. Therefore, AACE recommends that this treatment, as in all GH treatment, be individualized and carefully monitored. The patient and parents should make a well-informed decision by reviewing risks, benefits and costs.

December 2003