Low-dose deferasirox treatment in allogeneic hematopoietic cell transplantation survivors: Evaluation of iron overload by measuring liver iron concentration and non-transferrin-bound iron

Abstract

 Iron overload is a common complication of allogeneic hematopoietic cell transplantation (HCT); however, its management remains to be studied. We retrospectively analyzed the efficacy and safety of low-dose deferasirox treatment in four HCT survivors with iron overload and measured serum ferritin levels, liver iron concentrations (LIC), and non-transferrin-bound iron (NTBI) levels. Patients who had become transfusion-independent after HCT were treated using 10 mg/kg/day deferasirox. Their median age was 36.5 years (range, 27-39), and they had survived a median of 66 months (range, 27-101) after HCT. After a median of 23.5 months (range, 16-34) of deferasirox treatment, serum ferritin levels and LIC decreased in all patients, and NTBI decreased in three patients. The median ferritin levels, LIC, and NTBI levels decreased from 6135 (range, 3720-10,500) to 1782 ng/mL (range, 775-6840), 24.6 (range, 9.6-43.0) to 7.8 mg/g (range, 2.8-42.3), and 1.26 (range, 0.89-2.09) to 0.82 μmol/L (range, 0.64-1.54), respectively. Abnormal liver function tests improved in all patients after deferasirox treatment. On the other hand, all patients experienced an increase in serum creatinine levels. In conclusion, treatment with low-dose deferasirox might be an effective alternative for allogeneic HCT survivors with iron overload.