Transfusional iron burden and liver toxicity after bone marrow transplantation for acute myelogenous leukemia and hemoglobinopathies

• Published in: Pediatric Blood & Cancer 2008-02, Vol.50 (2), p.319-324
• Main Authors: Jastaniah, Wasil, Harmatz, Paul, Pakbaz, Zahra, Fischer, Roland, Vichinsky, Elliott, Walters, Mark C.
• Format: Article
• Language: English
• Subjects: Adolescent; Anemia, Sickle Cell - blood; Anemia, Sickle Cell - metabolism; Anemia, Sickle Cell - therapy; beta-Thalassemia - blood; beta-Thalassemia - metabolism; beta-Thalassemia - therapy; bone marrow transplant; Bone Marrow Transplantation - adverse effects; Child; Child, Preschool; Female; Humans; Iron - blood; Iron - metabolism; iron overload; Iron Overload - blood; Iron Overload - etiology; Iron Overload - metabolism; Leukemia, Myelogenous, Chronic, BCR-ABL Positive - blood; Leukemia, Myelogenous, Chronic, BCR-ABL Positive - metabolism; Leukemia, Myelogenous, Chronic, BCR-ABL Positive - therapy; Liver - metabolism; Liver Diseases - blood; Liver Diseases - etiology; Liver Diseases - metabolism; Longitudinal Studies; Male; phlebotomy; Retrospective Studies; transfusion; veno-occlussive disease; VOD
• ISSN: 1545-5009; 1545-5017; 1096-911X
• DOI: 10.1002/pbc.21260

Background While it is appropriate to treat transfusional iron overload to limit end‐organ injury after bone marrow transplantation (BMT) for β‐thalassemia major (TM), this approach after BMT for sickle cell disease (SCD) and hematological malignancies has not been studied. Procedure Fifteen children with SCD (n = 4), TM (n = 6), or acute myelogenous leukemia (AML, n = 5) underwent HLA‐identical sibling BMT between 2000 and 2003. Prospective evaluations of iron biomarkers were performed and the three groups were compared. Results The pre‐BMT duration and volume of RBC transfusions varied among the three groups, but baseline ferritin and liver iron concentration (LIC) were similar. In contrast, liver histology differed. Liver inflammation was present in four TM patients and portal fibrosis was observed in five TM and one SCD patient. Hepatic veno‐occlusive disease (VOD) developed in 5 of 15 patients. VOD was not associated with age, ferritin, ALT, or transfusions, but an association with liver inflammation and elevated LIC was suggested. Phlebotomy was performed in five patients after BMT. Changes in LIC were minimal in non‐phlebotomized patients (P = 0.02). Conclusion Iron biomarkers demonstrated significant iron overload before BMT in patients with malignant and non‐malignant disorders. However, iron overload was associated with liver inflammation and VOD primarily in TM patients. The clinical significance of iron overload in patients after BMT remains uncertain, but this is the first study to suggest that VOD may be associated with transfusional iron burden. Pediatr Blood Cancer 2008;50:319–324. © 2007 Wiley‐Liss, Inc.