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Treatment of dwarfism with recombinant human insulin-like growth factor-1 Ranke MB; Wolfle J; Schnabel D; Bettendorf MDtsch Arztebl Int 2009[Oct]; 106 (43): 703-9BACKGROUND: The growth hormone-IGF (insulin-like growth factor) system plays a central role in hormonal growth regulation. Recombinant human (rh) growth hormone (GH) has been available since the late 1980s for replacement therapy in GH-deficient patients and for the stimulation of growth in patients with short stature of various causes. Growth promotion by GH occurs in part indirectly through the induction of IGF-1 synthesis. In primary disturbances of IGF-1 production, short stature can only be treated with recombinant human IGF-1 (rhIGF-1). rhIGF-1 was recently approved for this indication but can also be used to treat other conditions. METHODS: Selective review of the literature on IGF-1 therapy, based on a PubMed search. RESULTS AND CONCLUSION: In children with severe primary IGF-1 deficiency (a rare condition whose prevalence is less than 1:10,000), the prognosis for final height is very poor (ca. 130 cm), and IGF-1 therapy is the appropriate form of pathophysiologically based treatment. There is no alternative treatment at present. The subcutaneous administration of IGF-1 twice daily in doses of 80 to 120 microg/kg accelerates growth and increases final height by 12 to 15 cm, according to current data. There is, however, a risk of hypoglycemia, as IGF-1 has an insulin-like effect. As treatment with IGF-1 is complex, this new medication should only be prescribed, for the time being, by experienced pediatric endocrinologists and diabetologists.|Dwarfism/*drug therapy[MESH]|Humans[MESH]|Insulin-Like Growth Factor I/*administration & dosage[MESH]|Recombinant Proteins/*therapeutic use[MESH] |
