Abstract
We administered recombinant IGF-I to 8 children with GHIS, due either to GH receptor deficiency (Laron syndrome, n=5) or growth-attenuating antibodies to GH (3 pts. with GH gene deletion). Their ages range from 3 to 11 years and they have been treated for 3 to 18 months (fig.). The dose of IGF-I (Genentech, Inc.) ranged between 80 and 120 mcg/kg. sc, twice daily. Two hours post-injection the patients consumed a snack or meal. Height velocity improved significantly in all but one patient (*). He had poor growth (2.6cm/yr) during the first 3 months, when he had intercurrent illnesses and poor nutrient intake. His growth velocity increased to 10.2cm/yr (error bar) during the second three months of treatment. Hypoglycemia has occured infrequently and only in the two youngest patients early in treatment. No adverse changes in biochemical profile have been observed. Increased height velocity has been accompanied by increased caloric intake and weight gain, a reduction in subcutaneous adipose tissue and catch-up growth of kidneys and spleen (by ultrasound), These data show that IGF-I stimulates linear growth by endocrine mechanisms and suggest: that chronic treatment with IGF-I Is likely to be a successful form of therapy for patients with GHIS.
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Backeljauw, P., Louis, E., Miras, M. et al. EFFECTS OF PROLONGED IGF-I TREATMENT IN CHILDREN WITH GROWTH HORMONE INSENSITIVITY SYNDROME (GHIS). Pediatr Res 33 (Suppl 5), S56 (1993). https://doi.org/10.1203/00006450-199305001-00315
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DOI: https://doi.org/10.1203/00006450-199305001-00315