Blood, Vol. 96 No. 1 (July 1), 2000:
pp. 76-79
Severity of iron overload in patients with sickle cell disease
receiving chronic red blood cell transfusion therapy
Paul Harmatz,
Ellen Butensky,
Keith Quirolo,
Roger Williams,
Linda Ferrell,
Thomas Moyer,
Daniel Golden,
Lynne Neumayr, and
Elliott Vichinsky
From the Departments of Gastroenterology, Hematology/Oncology, and
Pathology, Children's Hospital Oakland, Oakland, and the Department of
Pathology, University of California, San Francisco/Stanford Health
Care, San Francisco, CA; and the Division of Clinical Biochemistry and
Immunology, Mayo, Rochester, MN.
Chronic transfusion therapy is being used more frequently to
prevent and treat the complications of sickle cell disease. Previous studies have shown that the iron overload that results from such therapy in other patient populations is associated with significant morbidity and mortality. In this study we examined the extent of iron
overload as well as the presence of liver injury and the predictive
value of ferritin in estimating iron overload in children with sickle
cell disease who receive chronic red blood cell transfusions. A poor
correlation was observed between serum ferritin and the quantitative
iron on liver biopsy (mean 13.68 ± 6.64 mg/g dry weight;
R = 0.350, P = .142). Quantitative iron was
highly correlated with the months of transfusion
(R = 0.795, P < .001), but serum ferritin at
biopsy did not correlate with months of transfusion (R = 0.308, P = .200). Sixteen patients had
abnormal biopsies showing mild to moderate changes on evaluation of
inflammation or fibrosis. Liver iron was correlated with fibrosis score
(R = 0.50, P = .042). No complications were
associated with the liver biopsy. Our data suggest that, in patients
with sickle cell disease, ferritin is a poor marker for accurately
assessing iron overload and should not be used to direct long-term
chelation therapy. Despite high levels of liver iron, the associated
liver injury was not severe.
