Treatment of dwarfism with recombinant human insulin-like growth factor-1.
BACKGROUND
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.


CONCLUSIONS
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.
Ranke
Michael B
MB
Universitätsklinik für Kinder- und Jugendmedizin, Sektion Pädiatrische Endokrinologie, Tübingen. michael.ranke@med.uni-tuebingen.de
Wölfle
Joachim
J
Schnabel
Dirk
D
Bettendorf
Markus
M
D004392:Dwarfism / Q000188:drug therapy*; D006801:Humans; D007334:Insulin-Like Growth Factor I / Q000008:administration & dosage*; D011994:Recombinant Proteins / Q000627:therapeutic use*
D011994:Recombinant Proteins; D007334:Insulin-Like Growth Factor I