Recombinant growth hormone treatment in short patients with thalassemia major: Results after 24 and 36 months

Treatment with recombinant growth hormone (rhGH), 0.6 IU/kg/week s.c., prev iously successfully conducted for one year, was continued in 15 (Group A) a nd 8 (Group B) short thalassemia major patients with reduced GH reserve, fo r two and three years, respectively. In Group A, height for chronological a ge (Ht SDSCA) increased significantly (p=0.021) from the start of treatment , but the positive effect was only apparent because of the concomitant slig ht worsening of height for bone age (Ht SDSBA). Median Delta Ht SDSCA/Delta Ht SDSBA was <1.0 with respect to both the start (0.87) and the end of the first year of rhGH therapy (0.89). IGF-I levels increased significantly (p =0.043) compared with values both at the start and at the end of the first year of rhGH therapy. In Group B neither Ht SDSCA nor Ht SDSBA differed sta tistically from starting values, the former having a positive trend and the latter a negative one. Median <Delta>Ht SDSCA/Delta Ht SDSBA was 0.92 with respect to the start, and 0.94 with respect to the end of the second year. IGF-I levels increased significantly (p=0.043) with respect to starting va lues. Our data show that the encouraging results described from the first y ear of rhGH treatment did not persist during the second and third years, an d we conclude that this is because increase in bone age with continued trea tment is equal to, or slightly greater than the height age increase. We pro pose that patients with thalassemia major with short stature should receive rhGH treatment for only one year, and that more prolonged treatment should be reserved for selected adolescents who have psychological problems due t o shortness; for these patients growth acceleration could represent the mai n goal, even if this leads to a substantially unchanged or slightly decreas ed final height.