Growth Hormone Use in Children with Idiopathic Short Stature

Abstract

OBJECTIVE: To review the indication, pharmacology, pharmacokinetics, efficacy, and adverse effects of recombinant human growth hormone in children with idiopathic short stature (ISS). DATA SOURCES: A MEDLINE search (1966–December 2003) was performed using the key words human growth hormone, somatropin, Humatrope, normal children, somatrem, and idiopathic short stature. Food and Drug Administration Advisory Committee Meeting minutes were also reviewed. STUDY SELECTION AND DATA EXTRACTION: The data presented in this review were obtained from published literature, abstracts presented at scientific meetings, and information on file with the manufacturer. Additional articles from these sources were also identified. Current issues of pediatric and endocrinology journals were reviewed for the most recent articles. Articles only addressing the use of growth hormone in normal, healthy children were used. DATA SYNTHESIS: Somatropin is indicated for use in children with ISS. Studies have shown modest benefit to final height achieved and, at the doses used for ISS, there have been no adverse effects associated with somatropin. Many questions still exist, however, concerning the most appropriate age to initiate treatment and duration of treatment. There are also many ethical concerns surrounding patient selection criteria and potential for increased off-label use. CONCLUSIONS: Growth hormone has been found to have modest efficacy in improving final height in children with ISS. The specific patient population likely to achieve maximal benefit, optimal dosage regimens, and the long-term adverse effects of extended duration of therapy are unknown.