OBJECTIVE Despite regular transfusion and desferrioxamine treatment, growth failure is commonly seen in adolescent children with beta-thalassaemia major. The growth failure has been thought to be due to GH resistance rather than GH deficiency. We investigated the effect of GH on short non-GH deficient children with beta-thalassaemia. METHODS Recombinant human GH was given in a dose of 0.14 IU/kg/day subcutaneously in an open study. METHODS Fifteen prepubertal Chinese children with beta-thalassaemia major (ranging from 7.16 to 14.7 years in age) with height -1.5 SD or more below the population mean for age and a growth velocity of less than 5 cm/year were treated with growth hormone for one year. All children had peak GH response > 15mlU/l to insulin induced hypoglycaemia and normal thyroid function and adrenal reserve. METHODS Anthropometric measurements were performed every 3 months. Morning urine was tested twice weekly for glycosuria. Blood count, renal and liver function tests, fasting blood glucose, IGF-I and fructosamine levels were assessed at entry and every 3 months during treatment. Fasting insulin was measured before and after 3 and 12 months of GH treatment. Skeletal maturity was assessed before and after one year of treatment. RESULTS Treatment was stopped in two children after 6 months because of poor growth response and noncompliance with treatment and in one child at 9 months because of bone marrow transplantation. In the 13 children, the growth velocity increased from 3.6 +/- 0.7 cm/year to 8 +/- 1.2 cm/year after one year of GH treatment (P < 0.001). IGF-I was low before treatment (10.1 +/- 2.7 nmol/l), rising significantly to 15.8 +/- 4.8, 18.4 +/- 4.6, 19.3 +/- 6.4 and 21.9 +/- 7.5 nmol/l at 3, 6, 9 and 12 months of treatment (P < 0.005). The mean pretreatment bone age in the 13 children was 9.58 +/- 1.41 years and increased to 10.53 +/- 1.43 years after one year of treatment (delta BA/CA 0.95 +/- 0.3 years). None of the patients developed glycosuria or hypertension. There was no significant change in blood count, renal and liver function, thyroid function, fasting blood glucose or insulin concentrations during treatment. CONCLUSIONS Growth failure in these children with normal GH reserve and low serum IGF-I concentrations would suggest GH insensitivity. Supraphysiological doses of exogenous GH can cause a significant increase in serum IGF-I levels and a significant improvement in short-term growth of short children with beta-thalassaemia major.