Growth hormone secretion in polytransfused prepubertal patients with homozygous beta-thalassemia. Effect of long-term recombinant GH (recGH) therapy.

摘要

Growth was monitored in 133 male and 150 female North Sardinian prepubertal patients with homozygous beta-thalassemia in order to ascertain the incidence of GH deficiency (GHD) and the effects of long-term recombinant GH (recGH) treatment on growth velocity and bone maturation. A significant reduction in growth velocity and a fall in IGF-I levels was observed in 19 male and 16 female patients (12.3%). Their peak GH responses to GHRH (5.45+/-0.78 and 4.99+/-0.86 ng/ml) and clonidine administration (4.21+/-0.32 and 4.15+/-0.27 ng/ml in males and females, respectively) were markedly reduced with respect to control subjects (p<0.01). No statistically significant correlation was found between chronological age, number of blood units received, plasma ferritin levels and plasma IGF-I levels as well as with peak GH response to stimulation. Thalassemic patients with GHD had plasma ferritin levels (1382.44+/-160.34 and 1255.23+/-139.81 ng/ml in males and females, respectively) significantly lower than those recorded in the other patients (2848.94+/-283.61 and 3077.82+/-220.51 ng/ml). Patients with GHD were treated with recGH for an average period of 59 months (range 26-124). Treatment was able to restore growth and to increase significantly plasma IGF-I levels. Growth velocity at the end of the first yr of treatment was 6.78+/-1.21 and 6.11+/-0.85 cm/yr in males and females, respectively. Growth velocity values and plasma IGF-I levels remained significantly higher than basal values throughout the period of treatment. However, treatment was unable to normalize bone maturation since bone age values were always reduced with respect to chronological age. No incidence of side effects was observed. These data indicate that GHD, when present, is one but not the sole cause of delayed bone maturation and height deficiency in thalassemia.