title: Comparison of organ dysfunction in transfused patients with SCD or beta thalassemia.
abstract: Although it is life saving, transfusion therapy has resulted in the majority of sickle cell anemia and thalassemia patients being at risk for hemosiderosis-induced organ damage. It is unknown whether the complications of iron overload are affected by the underlying disease. In order to address this problem, we compared the prevalence of organ dysfunction in both groups of patients receiving chronic transfusion therapy (beta thalassemia, N = 30; sickle cell anemia, N = 43). Both groups had similar quantitative liver iron. Thalassemia patients had greater cardiac disease (20% vs. 0%), growth failure (27% vs. 9%), and endocrine failure (37% vs. 0%). The strongest predictors of combined endocrine and cardiac disease in multivariate analysis were duration of chronic transfusion (P = 0.03) and diagnosis (P = 0.03). Quantitative liver iron concentration on a single liver biopsy was not predictive of cardiac or endocrine injury. Viral hepatitis is the strongest predictor of hepatocellular damage (P = 0.009), while the development of liver fibrosis is more closely related to liver iron concentration (P = 0.04). In conclusion, sickle cell anemia and thalassemia differ in the prevalence of organ injury. This difference is related to the duration of iron exposure and the specific hemoglobinopathy. A prospective study with a larger number of subjects is needed to confirm the relationships between specific diagnosis, liver iron concentration over time, and organ dysfunction.
authors.affiliation: Department of Hematology/Oncology, Children's Hospital & Research Center at Oakland, CA 94609, USA.
authors.forename: Elliott
authors.initials: E
authors.lastname: Vichinsky
authors.forename: Ellen
authors.initials: E
authors.lastname: Butensky
authors.forename: Ellen
authors.initials: E
authors.lastname: Fung
authors.forename: Mark
authors.initials: M
authors.lastname: Hudes
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Theil
authors.forename: Linda
authors.initials: L
authors.lastname: Ferrell
authors.forename: Roger
authors.initials: R
authors.lastname: Williams
authors.forename: Leslie
authors.initials: L
authors.lastname: Louie
authors.forename: Phillip D K
authors.initials: PD
authors.lastname: Lee
authors.forename: Paul
authors.initials: P
authors.lastname: Harmatz
mesh_terms: D000293:Adolescent; D000755:Anemia, Sickle Cell / Q000628:therapy; D002908:Chronic Disease; D017707:Erythrocyte Transfusion / Q000009:adverse effects*; D005260:Female; D006801:Humans; D007249:Inflammation; D007501:Iron / Q000378:metabolism; D008099:Liver / Q000378:metabolism; D008297:Male; D012189:Retrospective Studies; D017086:beta-Thalassemia / Q000628:therapy*
chemical_list: D007501:Iron