RECOMBINANT-HUMAN-ERYTHROPOIETIN FOR LONG-TERM TREATMENT OF ANEMIA INPAROXYSMAL-NOCTURNAL HEMOGLOBINURIA

The long-term effects of recombinant human erythropoietin (rhEPO) admi nistration in two consecutive cases of paroxysmal nocturnal hemoglobin uria (PNH) with severe anemia are reported. In both patients, a 68-yea r-old woman and a 66-year-old man, a diagnosis of PNH was made on the basis of severe macrocytic anemia associated with hemoglobinuria, hemo siderinuria and positivity for the sucrose and Ham tests. Subcutaneous treatment with rhEPO, 150 U/Kg body weight daily, was followed in bot h cases by a progressive increase in hemoglobin concentrations, which there-after were maintained above 10 g/dL with lower doses of rhEPO an d without any relevant side effects for 32 and 29 months of continuous treatment, respectively. A clinical response was observed in spite of elevated baseline serum erythropoietin concentrations, appropriate to the degree of anemia in both patients. These results suggest that rhE PO may be appropriately and safely used in the long-term correction of anemia associated with PNH, and that the response to the pharmacologi c doses of rHEPO administered was not dependent on the level of endoge nous erythropoietin.