Growth Hormone in the Treatment of Children with Short Stature

Therapeutic use of growth hormone (GH) in the United States generally has been restricted to GH-deficient children who, with their families, agree to participate in research. The limited supply and the considerable expense of commercially prepared GH have restricted the number of children who have received such therapy. As the medical community and public become more aware of the therapeutic potential of GH, practicing physicians are faced with mounting pressure to prescribe GH for short children who are not GH deficient. Furthermore, the promise that abundant supplies of biosynthetic GH prepared by recombinant DNA methods will soon be available adds to the fever for uncontrolled experimentation.

Twenty-five years of experience indicate that therapeutic replacement of GH is safe for GH-deficient children. Nevertheless, using GH in the treatment of children who are not deficient in GH should not be engaged in lightly, as GH is a potent metabolic agent that might have significant side effects. Because there is so little experience with the therapeutic use of GH at the pharmacologic doses that may be required to accelerate growth in non-GH-deficient children, information on side effects is limited. It is appropriate, therefore, to review (1) the accepted indications for the therapeutic use of GH (GH-deficient children), (2) the disorders in which GH has been shown to lack beneficial effect, (3) the issues to be considered in selecting for therapy patients who do not appear to have classic GH deficiency, and (4) the side effects that might occur when GH is administered as a pharmacologic agent.

TREATMENT OF GROWTH HORMONE DEFICIENT CHILDREN

Growth failure due to GH deficiency is the only universally accepted therapeutic indication for GH treatment.