Use of recombinant human growth hormone in children with thalassemia.

Background: Growth failure occurs in children with chronic anemias and, in particular, in approximately 30% of those with thalassemia.

Methods: We assessed recombinant human growth hormone (rhGH) use in a large cohort of children with thalassemia enrolled in the Pfizer International Growth Study Database (KIGS).

Results: We identified 147 short children with thalassemia who were treated with rhGH in KIGS. Of these, approximately 40% had a primary diagnosis of GH deficiency (GHD). They had low birth weight, short parents, reduced genetic height potential, low insulin-like growth factor I levels and delayed bone age. Treatment with rhGH for 1 year resulted in a significantly increased growth rate regardless of underlying GH or pubertal status. Although the resultant growth rates for thalassemic children were significantly higher than at baseline, they were less than those seen in similarly treated short children with or without GHD.

Conclusions: GH testing should be performed in short thalassemic children, and those with GHD should be treated with rhGH. The value of rhGH therapy in short thalassemic children without GHD is less clear-cut and requires further study regarding final height outcome.