A prospective study of iron overload management in allogeneic hematopoietic cell transplantation survivors.

We report the results of a single-center, prospective evaluation for iron overload and subsequent treatment in 147 adult allogeneic hematopoietic cell transplantation (HCT) recipients who survived beyond 1 year after transplantation. Patients were screened by serum ferritin level; those with ferritin >1000 ng/mL underwent liver R2 magnetic resonance imaging to estimate liver iron concentration (LIC; normal < or =1.8 mg/g). Patients with significant iron overload (defined as LIC > or =5 mg/g), based on physician and patient preference, were offered observation only, phlebotomy, or enrollment in a pilot study of deferasirox. Sixteen patients had significant iron overload. Their median age was 51 years (range, 29-64 years), and they had survived a median of 21 months (range, 12-114 months). All 16 patients were transfusion-independent at study enrollment. Five patients received no treatment (median LIC, 6.4 mg/g; range, 5.1-28.3 mg/g), 8 underwent phlebotomy (median LIC, 13.1 mg/g; range, 7.8-43.0 mg/g), and 3 received daily deferasirox 20 mg/kg/day orally for 6 months (LIC, 6.3, 9.0, and 19.9 mg/g). Two patients had abnormal liver function tests, and 1 patient each had cirrhosis and unexplained congestive heart failure; all 4 of these patients underwent phlebotomy. Follow-up serum ferritin concentrations decreased spontaneously in 4 patients in the observation-only arm. Phlebotomy was generally well tolerated. Deferasirox also was well tolerated and led to decreased LIC after 6 months of therapy in all 3 patients. Phlebotomy is feasible in the majority of allogeneic HCT recipients who have survived for > or =1 year after HCT and have significant iron overload. Although the number of subjects is small, deferasirox may be a safe and effective alternative for allogeneic HCT survivors with iron overload who cannot undergo phlebotomy. Copyright 2010 American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.