Growth hormone treatment in short children with beta-thalassemia major

The effect of one year recombinant human growth hormone (rhGH) treatment on growth rate and bone age was studied in ten short prepubertal children wit h P-thalassemia major (age range 7.10-12.03 yr) with normal GH response to provocative stimuli, rhGH was given subcutaneously every day in a dose of 2 8 IU/m(2)/week. In the 10 children who completed 12 months of treatment the growth velocity increased from 4.22+/-0.81 cm/yr (-1.38+/-0.80 SDS for CA) to 7.61+/-1.16 cm/yr (+2.27+/-1.64 SDS for CA), IGF-I was low before treat ment, 138.3+/- 38.9 ng/ml, and rose significantly to 232.2+/-122.1, 243.2+/ -98.4 and 227.5+/-86.2 at 3, 6 and 12 months post-treatment, respectively ( p<0.01). Bone maturation was accelerated in proportion to the increase in c hronological age. The mean pretreatment bone age in the ten children was 8. 20+/- 1.97 and increased to 9.55+/-1.80 yr after one year of treatment. Our data demonstrate that GH treatment of thalassemic children with normal GH reserve and low serum IGF-I concentrations with supraphysiological doses of rhGH for one year can cause a significant increase in serum IGF-I levels a nd growth velocity, but it remains to be elucidated whether long-term admin istration will affect the final height.