title: Recombinant human insulin-like growth factor-I therapy for children with growth disorders.
abstract: Until recently, the only possible therapy available for treatment of children with significant short stature was recombinant human growth hormone (rhGH). However, recombinant human insulin-like growth factor-I (rhIGF-I) has now become commercially available as a therapeutic option to treat children of short stature caused by severe primary IGF-I deficiency, defined as: height standard deviation score (SDS) less than or equal to -3.0, basal IGF-I SDS less than or equal to -3.0, and normal or elevated levels of GH. Published data demonstrate that rhIGF-I therapy in patients with primary IGF-I deficiency accelerates growth significantly during the first year of treatment, but progressive attenuation is likely in subsequent years. The growth response to rhIGF-I is neither as intense nor as well sustained as the growth response to rhGH among children with GH deficiency. Despite increasing interest in the possibility for broader use of rhIGF-I for growth promotion, especially in children with idiopathic short stature (ISS), it is necessary to wait for studies assessing the efficacy and safety of rhIGF-I therapy in this condition. In this particular population (ISS patients), the combination of rhIGF-I and rhGH, compared with either hormone used alone, may have theoretical advantages. Hypoglycemia has been the most common side effect reported with use of rhIGF-I and is reasonably controlled with adequate food intake. Most of the other (long-term) adverse effects appear to be related to hyperstimulation of lymphoid tissue growth. Little is known about the long-term effects of IGF-I therapy in growing children, but caution and long-term, controlled, prospective trials of rhIGF-I-treated children and adolescents are needed.
authors.affiliation: Pediatric Endocrinology, National Children's Hospital, San José, Costa Rica. erichmondp@hnn.sa.cr
authors.forename: Erick J
authors.initials: EJ
authors.lastname: Richmond
authors.forename: Alan D
authors.initials: AD
authors.lastname: Rogol
mesh_terms: D001827:Body Height / Q000187:drug effects; D002648:Child; D006130:Growth Disorders / Q000188:drug therapy* / Q000503:physiopathology; D006801:Humans; D007334:Insulin-Like Growth Factor I / Q000009:adverse effects / Q000627:therapeutic use*; D036341:Intercellular Signaling Peptides and Proteins / Q000627:therapeutic use; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use
chemical_list: D036341:Intercellular Signaling Peptides and Proteins; D011994:Recombinant Proteins; C065700:myotrophin; D007334:Insulin-Like Growth Factor I