[Effects of rHuEPO on aplastic anemia: results of a phase II clinical study]

The safety and efficacy of recombinant human erythropoietin (epoetin alpha) were investigated in adult aplastic anemia patients whose hemoglobin (Hb) concentration was less than 10g/dl. Epoetin alpha was given subcutaneously every day at a dose of 3,000IU/body for two weeks, and the dosage was increased to 6,000IU, 12,000IU and 24,000IU every two weeks when the increment of Hb was insufficient. In cases in whom Hb concentration increased by more than 1g/dl or whose transfusion requirements reduced to less than 50%, treatment was judged to be effective. The whole rate of efficacy was 34.5% (10/29). Response to epoetin alpha treatment was better in patients whose symptoms were relatively mild. Mild cases responded to the treatment with 6,000IU/body/day, although a dosage of 24,000IU/body/day was required in moderate or severe cases. Neither serious adverse effect nor abnormal laboratory findings were observed. These results suggest that high dose subcutaneous epoetin alpha treatment is effective for the aplastic anemia in terms of increasing Hb concentration and reducing blood transfusions.