USE OF RECOMBINANT ERYTHROPOIETIN IN THALASSEMIC PATIENTS ON DIALYSIS

We studied the therapeutic benefit of recombinant human erythropoietin (rHuEPO) in dialysis patients with thalassemia minor. Four of the 40 randomly selected patients (22 on hemodialysis [HD], 18 on continuous ambulatory peritoneal dialysis [CAPD]) were identified to be thalassemic prior to a trial of rHuEPO (α-thalassemia trait in three and β-thalassemia minor in one). All patients were initially treated with rHuEPO at a dose of 100 ± 25 U/kg/wk subcutaneously depending on the hemoglobin level. EPO injections were continued for 16 weeks with further adjustments of the doses according to the hemoglobin level increases attained. All nonthalassemic patients reached a target hemoglobin of 10 g/dL at week 16, with an average maintenance dose of 120 ± 7.8 U/kg/wk, but the hemoglobin was increased by only 1 g/dL in the thalassemic patients receiving 175 U/kg/wk. Following cessation of rHuEPO therapy for 6 weeks, all four thalassemic patients and 18 randomly selected nonthalassemic patients received a fixed dose of rHuEPO 4,000 U/wk (equivalent to 80 U/kg/wk) for 16 weeks. The hemoglobin remained unchanged in the thalassemic patients, but a progressive and significant increase of hemoglobin was observed in the nonthalassemic patients. At the last phase of the study, the thalassemic patients received rHuEPO at a dose of 100 or 125 U/kg/wk with 4-weekly increments of 25 U/kg/wk until their hemoglobin reached 10 g/dL. One patient developed uncontrolled hypertension with a dose of 150 U/kg/wk, and one reached the target hemoglobin at a dose of 200 U/kg/wk. The remaining two patients maintained their hemoglobin at approximately 9 g/dL at a dose of 250 U/kg/wk; further increments were withheld because of hypertension in one and renal transplantation in the other. Our findings suggest the erythropoietic response to rHuEPO in uremic patients with thalassemia minor is promising, but a higher dose is required and the clinical response is often delayed. Furthermore, administration of a higher dose of rHuEPO is not uncommonly limited by the rHuEPO-related complications. © 1992, National Kidney Foundation. All rights reserved.. All rights reserved.