Thirty-nine children with Fanconi
aplastic anemia (FAA) have been followed up in our center between January 2008 and November 2010. Eight of these children (20%) with a transfusional
iron overload had been undergoing
deferasirox treatment during the study period. In the English literature, transfusional
iron overload and the use of an
iron chelator in children with FAA has not yet been evaluated. Here, we have presented the effectivity and tolerability of
deferasirox in children with FAA and a transfusional
iron overload. Before the
deferasirox treatment, the mean serum
ferritin level was 3377 ± 2200 ng/mL. After a mean 13.6-month
treatment duration, the mean
ferritin level decreased to 2274 ± 1300 ng/mL (P<0.05). In our series, 3 patients had renal and 3 had hepatic toxicity during the treatment. Two patients had
peliosis hepatis and 2 had congenital renal abnormalities before the treatment. There may be differences in the side-effect profiles of
deferasirox treatment in patients with FAA. In our series, despite the low number of cases, nephrotoxicity and hepatotoxicity were common side effects instead of gastrointestinal disturbances reported in other studies.
Deferasirox is an oral, easily applicable, and effective
iron chelator; baseline hepatotoxicity and nephrotoxicity may increase the development of toxic side effects in children with FAA. Patients with FAA receiving
deferasirox treatment should be followed up closely for these side effects.