Abstract
30 patients with GH insensitivity syndrome (GHIS) (Laron syndrome) and 2 patients with GH gene deletion were included in a multicentre trial of recombinant human IGF-1 (rhIGF-1). The GHIS patients, from 9 European countries and Australia, were 15 of each sex, aged 3.7 - 22.9 y and 5 were pubertal. Clinical and endocrine features of GHIS were (median, range): Height SDS −6.7 (−9.1 to −3.2), basal GH 17 mU/L (2.4 - 208), IGF-1 21 μg/L (<20 - 69) and no response of IGF-1 to four days of GH (GenotropinR 0. IIU/Kg/day). At baseline IGF-2 was 168 H-g/L (43 - 295) and 19 had no measurable GH binding protein in serum. The GH gene deletion patients were a girl aged 14.2 y and a boy aged 16.7 y. 13 patients were initially started on rhIGF-1 40 μg/KG BWt s.c. bd, 7 increasing to 120 μg/Kg BWt bd at 3-6 months because of poor response. 17 GHIS and 2 GH gene deletion patients started on 120 μg/Kg BWt bd.
Interestingly, despite an anabolic effect of rhIGF-1, serum levels of IGFBP-3, the principal carrier protein of IGF-1, did not change during treatment; basal was 0.69 mg/L (0.13 - 1.59), after 6 months therapy 0.46 mg/L (0.22 - 1.11). Hypoglycaemia (B.S. <3.0 mmol/L), with or without symptoms, occurred in 17 patients, 2 having consulsions. 12 patients reported mild or moderate headache, 1 severe with vomiting and bilateral papilloedema which resolved after discontinuation of rhIGF-1. 2 patients reported pain consistent with renal colic. 1 patient developed an unilateral Bell's palsy which resolved within 14 days.
CONCLUSIONS: rhIGF-1 in a dose of 40 - 120 μg/Kg BWt bd increased linear growth in children with GHIS and GH gene deletion. Serum IGFBP-3 did not change. This may contribute to hypoglycaemia, presumably related to high levels of free IGF-1. A growth promoting effect of this new therapeutic agent has been clearly demonstrated.
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Savage, M., Wilton, P., Ranke, M. et al. THERAPEUTIC RESPONSE TO RECOMBINANT IGF-1 IN THIRTY TWO PATIENTS WITH GROWTH HORMONE INSENSITIVITY. Pediatr Res 33 (Suppl 5), S5 (1993). https://doi.org/10.1203/00006450-199305001-00018
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DOI: https://doi.org/10.1203/00006450-199305001-00018
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