Abstract 26

In most Hereditary Spherocytosis (HS) affected newborns, transient transfusion need correlates with a poor erythroid response to anemia, as illustrated by reticulocyte counts inappropriately low for the severity of anemia. While 50-80% of anemic HS newborns are transfused one or several times during the first year of life, less than 10-20% will need further transfusion support. This transient character of transfusion need led us to wonder whether, likewise anemic premature infants, anemic HS infants could benefit from recombinant human erythropoietin (rHu-Epo) therapy. During the years 1996-98, 16 anemic HS patients age ranging from 16 to 119 days, were prescribed rHu-Epo treatment. rHu-Epo initial doses, ranging from 500 to 1000 IU/kg/week, were administered subcutaneously 2 or 3 times a week. Discontinuation was decided either when patients had reached a steady state of compensated hemolysis or because of treatment failure with doses ≥ 1000 IU/kg/week. Ten patients were classified as good responders because patients with no transfusion need under treatment and after discontinuation. In 3 patients, classified as partial responders, a single red blood cell transfusions was prescribed during the course of rHu-Epo treatment. A failure of treatment was observed in 3 patients. In these three severe forms, further transfusion were needed during treatment as well as after discontinuation of therapy. In all patients the treatment was well tolerated with no side-effect. Our experience shows that the use of rHu-Epo can be a valuable alternative to RBC transfusion but implies an early diagnosis and a close follow-up in order to determine in due time the patients eligible for therapy.