Administration of Erythropoietin to Patients with P-thalassemia Intermedia: a Preliminary Trial

Patients with thalassemia major are considered to have high erythropoietin (Epo) activity.' However, Hammond et a1 reported that thalassemic patients had much less serum Epo activity compared with patients with hypoplastic anemia.* Determination of serum Epo levels in 32 patients from our clinic with homozygous p-thalassemia major and intermedia showed that Epo activity was not significantly higher than in normal controls except for the groups of minimally transfused infants who had higher levels, similar to those found in patients with severe aplastic anemia.3 A low serum Epo level for the degree of the anemia was also found in patients with sickle cell anemia.4 Recombinant human Epo (rhuEpo) has been shown to significantly increase red blood cells (RBCs), hemoglobin (Hb), and hematocrit (Hct) in patients with chronic renal failure, both in those who were maintained on hemodialysis and in those not requiring dialy~is.~ ' These observations may suggest that rhuEpo could be effective in patients with p-thalassemia intermedia and may result in an increase of 1 to 3 g of Hb, thus improving the quality of life without causing symptoms due to accelerated hematopoiesis. It is also possible that rhuEpo may specifically stimulate synthesis of fetal Hb (HbF).' Three patients with @-thalassemia intermedia were studied (two Arabs and a Kurdish Jew, Table 1). rhuEpo (EPREX) kindly supplied by Cilag AG International (SchafThausen, Switzerland) was administered in an initial dose of 1,000 Uikg intravenously twice daily, 1 day a week for 2 weeks. One hundred and ten days later, a second course of rhuEpo in a dose of 500 U k g was administered twice a week for 6 weeks and 1,000 U k g were administered twice a week for 3 more weeks (Fig 1). Globin chain