title: A moderate transfusion regimen may reduce iron loading in beta-thalassemia major without producing excessive expansion of erythropoiesis.
abstract: BACKGROUND
  Hypertransfusion with a baseline hemoglobin of 10 to 12 g per dL is still considered by many to be the mainstay of conservative therapy for beta-thalassemia major. However, this regimen is frequently associated with manifestations of transfusion iron overload, despite regular chelation therapy with subcutaneous desferoxamine.
  
  
  METHODS
  To verify whether a transfusion regimen with a target pretransfusion hemoglobin level between 9 and 10 g per dL can allow a significant reduction in blood consumption, while still effectively suppressing erythropoiesis, the records were reviewed of 32 beta-thalassemia major patients, who were maintained at a pretransfusion hemoglobin of 11.3 +/- 0.5 g per dL between 1981 and 1986. These patients were switched at the beginning of 1987 to a transfusion regimen with pretransfusion hemoglobin of 9.4 +/- 0.4 g per dL. The degree of erythroid marrow activity was evaluated in these patients and in 32 subjects with beta-thalassemia intermedia through the simple measurement of serum transferrin receptor.
  
  
  RESULTS
  After the adoption of the moderate transfusion regimen, transfusion requirements decreased from 137 +/- 26 to 104 +/- 23 mL per kg per year of red cells (p < 0.0001), and mean serum ferritin decreased from 2448 +/- 1515 to 1187 +/- 816 micrograms per L (p < 0.0001), with one-half of patients achieving serum ferritin levels lower than 1000 micrograms per L. The proportion of patients having spontaneous pubertal development increased significantly (p < 0.01), as a result of less iron-related gonadotropin insufficiency. At the lower pretransfusion hemoglobin, erythroid marrow activity did not exceed two to three times normal levels in most subjects.
  
  
  CONCLUSIONS
  As compared with hypertransfusion, moderate transfusion may allow more effective prevention of iron loading, with higher likelihood of spontaneous pubertal development and without producing excessive expansion of erythropoiesis.
authors.affiliation: Department of Internal Medicine, University of Pavia, Italy.
authors.forename: M
authors.initials: M
authors.lastname: Cazzola
authors.forename: C
authors.initials: C
authors.lastname: Borgna-Pignatti
authors.forename: F
authors.initials: F
authors.lastname: Locatelli
authors.forename: L
authors.initials: L
authors.lastname: Ponchio
authors.forename: Y
authors.initials: Y
authors.lastname: Beguin
authors.forename: P
authors.initials: P
authors.lastname: De Stefano
mesh_terms: D000293:Adolescent; D001803:Blood Transfusion; D002648:Child; D003676:Deferoxamine / Q000627:therapeutic use; D004920:Erythropoiesis / Q000502:physiology*; D006128:Growth; D006454:Hemoglobins / Q000032:analysis; D006801:Humans; D019190:Iron Overload / Q000628:therapy*; D011990:Receptors, Transferrin / Q000097:blood; D017262:Siderophores / Q000627:therapeutic use; D065227:Transfusion Reaction; D018754:Ventricular Dysfunction / Q000209:etiology; D017086:beta-Thalassemia / Q000097:blood / Q000378:metabolism / Q000628:therapy*
chemical_list: D006454:Hemoglobins; D011990:Receptors, Transferrin; D017262:Siderophores; D003676:Deferoxamine