title: Recombinant erythropoietin therapy as an alternative to blood transfusions in infants with hereditary spherocytosis.
abstract: BACKGROUND
  In hereditary spherocytosis, erythropoiesis has been described as 'sluggish' during the first months of life. The lack of appropriate erythropoietic response to compensate for increased red cell destruction necessitates blood transfusions in 70-80% of hereditary spherocytosis-affected infants during their first year of life. After this period, less than 30% require regular transfusion support. This transient requirement for transfusion led us to wonder whether anemic hereditary spherocytosis infants, like anemic premature infants, could benefit from recombinant erythropoietin therapy (rHu-Epo).
  
  
  METHODS
  In 16 hereditary spherocytosis infants (age range 16-119 days) with severe anemia, a compassionate open preliminary study was performed. rHu-Epo treatment (1000 IU/kg/week) was instituted together with iron supplementation. Hemoglobin values and reticulocyte counts were repeatedly assessed.
  
  
  RESULTS
  In 13 out of 16 infants, prompt increases in reticulocyte counts were noted after the first week of treatment with 1000 IU/kg/week of rHu-Epo. During treatment with Epo these infants maintained clinically acceptable levels of hemoglobin and did not require blood transfusions. As the infants grew and began to mount an adequate erythropoietic response, the rHu-Epo dose could be tapered and the treatment could be discontinued before the age of nine months.
  
  
  CONCLUSIONS
  Epo treatment in most hereditary spherocytosis infants appears to be effective in the management of anemia and could serve as a valuable alternative to packed RBC transfusions.
authors.affiliation: Laboratoires d'Hématologie, de Biochimie, département de Pédiatrie et Faculté de Médecine Paris-Sud, Hôpital Bicêtre, Le Kremlin-Bicêtre, Assistance Publique-Hôpitaux de Paris, France. gilbert.tchernia@bct.ap-hop-paris.fr
authors.forename: G
authors.initials: G
authors.lastname: Tchernia
authors.forename: F
authors.initials: F
authors.lastname: Delhommeau
authors.forename: S
authors.initials: S
authors.lastname: Perrotta
authors.forename: T
authors.initials: T
authors.lastname: Cynober
authors.forename: B
authors.initials: B
authors.lastname: Bader-Meunier
authors.forename: B
authors.initials: B
authors.lastname: Nobili
authors.forename: P
authors.initials: P
authors.lastname: Rohrlich
authors.forename: J L
authors.initials: JL
authors.lastname: Salomon
authors.forename: S
authors.initials: S
authors.lastname: Sagot-Bevenot
authors.forename: E M
authors.initials: EM
authors.lastname: del Giudice
authors.forename: J
authors.initials: J
authors.lastname: Delaunay
authors.forename: D
authors.initials: D
authors.lastname: DeMattia
authors.forename: P O
authors.initials: PO
authors.lastname: Schischmanoff
authors.forename: N
authors.initials: N
authors.lastname: Mohandas
authors.forename: A
authors.initials: A
authors.lastname: Iolascon
mesh_terms: D001803:Blood Transfusion; D017707:Erythrocyte Transfusion; D004921:Erythropoietin / Q000627:therapeutic use*; D005260:Female; D018392:Genomic Imprinting; D005865:Gestational Age; D006454:Hemoglobins / Q000187:drug effects / Q000378:metabolism*; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007501:Iron / Q000627:therapeutic use; D008297:Male; D011994:Recombinant Proteins; D017701:Reticulocyte Count; D013103:Spherocytosis, Hereditary / Q000097:blood / Q000235:genetics / Q000628:therapy*
chemical_list: D006454:Hemoglobins; D011994:Recombinant Proteins; D004921:Erythropoietin; D007501:Iron