title: Deferasirox therapy in children with Fanconi aplastic anemia.
abstract: 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.
authors.affiliation: Department of Pediatric Hematology, Ankara Children's Hematology and Oncology Hospital, Diskapi, Ankara/Turkey.
authors.forename: Bahattin
authors.initials: B
authors.lastname: Tunç
authors.forename: Betul
authors.initials: B
authors.lastname: Tavil
authors.forename: Neslihan
authors.initials: N
authors.lastname: Karakurt
authors.forename: Nese
authors.initials: N
authors.lastname: Yarali
authors.forename: Fatih Mehmet
authors.initials: FM
authors.lastname: Azik
authors.forename: Abdurrahman
authors.initials: A
authors.lastname: Kara
authors.forename: Vildan
authors.initials: V
authors.lastname: Culha
authors.forename: Serdar
authors.initials: S
authors.lastname: Ozkasap
mesh_terms: D000284:Administration, Oral; D000293:Adolescent; D001565:Benzoates / Q000008:administration & dosage* / Q000009:adverse effects; D001803:Blood Transfusion; D002648:Child; D002675:Child, Preschool; D000077588:Deferasirox; D005199:Fanconi Anemia / Q000097:blood / Q000188:drug therapy*; D005260:Female; D005293:Ferritins / Q000378:metabolism; D005500:Follow-Up Studies; D006801:Humans; D007502:Iron Chelating Agents / Q000008:administration & dosage* / Q000009:adverse effects; D019190:Iron Overload / Q000097:blood / Q000188:drug therapy / Q000209:etiology; D008297:Male; D014230:Triazoles / Q000008:administration & dosage* / Q000009:adverse effects
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D000077588:Deferasirox