Treatment with biosynthetic growth hormone in short thalassemic patients with impaired growth hormone secretion

Objective - Impairment of linear growth is a common clinical feature in patients with β-thalassaemia major. Although growth hormones secretion appears to be normal in many short thalassaemic patients, it proves to be deficient in some of them. In these cases, administration of biosynthetic growth hormone seems justified. The aim of this study was to evaluate the effect of such treatment in a group of patients with β-thalassaemia major presenting with growth failure and impairment of growth hormone secretion. Design - Recombinant human growth hormone, 0.6 U/kg body weight per week, given subcutaneously in three divided doses, was administered for 12 months. Patients - Eight prepubertal patients with β-thalassaemia major, presenting with severe growth retardation and impaired growth hormone secretion in response to provocative stimuli (insulin-induced hypoglycaemia, L-dopa and growth hormone-releasing hormone), were investigated. Measurements - Height and pubertal stage of the patients, as well as plasma levels of insulin-like growth factor I, were determined before, during and after biosynthetic growth hormone treatment. Results - During the first 6 months of therapy, a significant increase of growth velocity was observed, from a mean pretreatment value of 2.1 ± 0.45 cm/year to a value of 4.8 ± 0.66 cm/year (P < 0.002). Mean growth rated at 12 months (4.1 ± 0.50 cm/year), though slightly decreased in comparison to that recorded at 6 months, was still significantly higher than basal (P < 0.001). A significant increase in plasma levels of insulin-like growth factor I was recorded during treatment (2.82 ± 0.47 vs 0.96 ± 0.22 U/ml, P < 0.005). No side-effects, adverse reactions or alterations in routine laboratory examinations ensued during or after therapy. Conclusions - It appears from these data that biosynthetic growth hormone administration is worth serious consideration in patients with β-thalassaemia major presenting growth retardation and impaired growth hormone secretion.