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Recombinant human insulin-like growth factor-I therapy for children with growth disorders

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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.

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Correspondence to Erick J. Richmond.

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Richmond, E.J., Rogol, A.D. Recombinant human insulin-like growth factor-I therapy for children with growth disorders. Adv Therapy 25, 1276–1287 (2008). https://doi.org/10.1007/s12325-008-0124-9

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