A moderate transfusion regimen may reduce iron loading in β-thalassemia major without producing excessive expansion of erythropoiesis

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 β-thalassemia major. However, this regimen is frequently associated with manifestations of transfusion iron overload, despite regular chelation therapy with subcutaneous desferoxamine. STUDY DESIGN AND 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 β-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 β-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 μg per L (p<0.0001), with one-half of patients achieving serum ferritin levels lower than 1000 μg 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. CONCLUSION: 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