Recombinant human growth hormone treatment in children with thalassemia major.

Background: To evaluate the growth hormone reserve and the growth hormone response to recombinant human growth hormone (GH) in prepubertal thalassemic children with growth retardation. Methods: Twenty thalassemic patients with short stature and delayed bone age were studied. Patients were randomized into GH-treated (n=10) and non-GH treated (control; n=10) groups. The GH-treated group received recombinant human (rh)-GH (Genotropin) at the dose of 0.7 IU/kg per week for 12 months. Results: There was a significant discordance between GH response to pharmacologic stimuli and physiological secretion of GH/GHRH testing. Following the administration of rhGH, growth velocity increased from 2.47~0.48 cm/year to 6.27~0.76 cm/year (P=0.005), whereas there was not a similar change in the non-GH-treated group. The height velocities of the two groups during the 1 year follow-up period were significantly different (6.27~0.76 vs 3.99~0.34 cm/year; P=0.025). There were significant differences between the height velocity improvements and height velocity standard deviation scores of the two groups as well. Conclusion: The present study has demonstrated that rhGH is a safe and efficacious mode of treatment in thalassemic children.