ESPE Abstracts

Background: Laron syndrome is an autosomal recessive disease caused by molecular defect in GH receptor gene. The patients have severe growth retardation and clinical features of GHD with low IGF-1, high basal GH and failed response to IGF-1 stimulation test. The only proved treatment is daily recombinant IGF-1 administration which showed improvement in linear growth.

Aim: To describe the largest group of patients with laron syndrome Saudi Arabia and asses the efficacy of IGF-1 treatment and complication.

Methodology: We conduct a retrospective chart review to all patients with laron syndrome who are receiving IGF-1 treatment at dosage of 100 mg /kg/dose +/- 20 mg BID in king Faisal specialist hospital and research center from 1998 till 2020. Initial height, basal GH, response to treatment and improvement will be described for every patient currently receives treatment and for those who already completed their treatment. 27 patients with GHIS followed since early childhood were included in the study.

Result: During the first year of therapy a substantial increase in linear growth velocity was witnessed, from a mean 3.4 to 6.5cm/year with the mean difference of 3.1cm (p < 0.0001). In the second year the growth velocity was lower, but still suggestively higher than before treatment, from 3.4 to 5cm/year with mean difference of 1.6cm (P:0.0015). Long term follow-up over 10 years presented an increase in growth velocity compared to the baseline, but changes were statistically significant in the first 5 years. However, weight SDS was expressively greater throughout the treatment as well. Age at starting of the therapy had no particular impact on the results except for the fact that the patient had stayed longer on the treatment.