Use of Hydroxyurea and Recombinant Erythropoietin In Management of Homozygous 0 Thalassemia

Summary: This report describes the sustained response of an Iranian girl with homozygous 0 thalassemia (IVS-II-1G→A) to hydroxyurea (HU) and recombinant erythropoietin (rEPO). Since the start of this regimen 7 years ago, she has been transfu- sion-independent and her hemoglobin is maintained between 9.5-11.0 gm/dL. She is maintaining consistent growth around the 10th percentile for age and enjoys a good quality of life. She has not had any therapy-related adverse effects. This experience sug- gests that therapy with HU and rEPO may be useful long-term in some patients with thalassemia.