title: Efficacy and safety of deferasirox estimated by serum ferritin and labile plasma iron levels in patients with aplastic anemia, myelodysplastic syndrome, or acute myeloid leukemia with transfusional iron overload.
abstract: BACKGROUND
  Patients receiving red blood cell (RBC) transfusions are at risk of iron overload, which can cause significant organ damage and is an important cause of morbidity and mortality.
  
  
  METHODS
  This study was an open-label, single-arm, prospective clinical study to evaluate the efficacy and safety of deferasirox (DFX) in patients with aplastic anemia (AA), myelodysplastic syndrome (MDS), or acute myeloid leukemia (AML). Patients with serum ferritin levels of at least 1000 ng/mL and ongoing transfusion requirements were enrolled. DFX was administered for up to 1 year. A total of 100 patients were enrolled.
  
  
  RESULTS
  Serum ferritin levels decreased significantly following treatment (from 2000 to 1650 ng/mL, p = 0.004). The median absolute reduction in serum ferritin levels was -65 ng/mL in AA (p = 0.037), -647 ng/mL in lower-risk MDS (MDS-LR; p = 0.007), and -552 ng/mL in higher-risk MDS (MDS-HR)/AML (p = 0.482). Mean labile plasma iron (LPI) levels decreased from 0.24 μmol/L at baseline to 0.03 μmol/L at 1 year in all patients (p = 0.036). The mean LPI reduction in each group was -0.17 μmol/L in AA, -0.21 μmol/L in MDS-LR, and -0.30 μmol/L in MDS-HR/AML. Gastrointestinal disorders were commonly observed among groups (16.0%). DFX was temporarily skipped for adverse events in seven patients (7.0%) and was permanently discontinued in 11 patients (11.0%).
  
  
  CONCLUSIONS
  DFX reduced serum ferritin and LPI levels in patients with transfusional iron overload. Despite the relatively high percentage of gastrointestinal side effects, DFX was tolerable in all subgroups.
authors.affiliation: Department of Hemato-Oncology, Inje University Haeundae Paik Hospital, Busan.
authors.forename: Il-Hwan
authors.initials: IH
authors.lastname: Kim
authors.affiliation: Department of Hematology/Oncology, Kyungpook National University Hospital, Daegu.
authors.forename: Joon-Ho
authors.initials: JH
authors.lastname: Moon
authors.affiliation: Department of Hemato-Oncology, Inje University Haeundae Paik Hospital, Busan.
authors.forename: Sung-Nam
authors.initials: SN
authors.lastname: Lim
authors.affiliation: Department of Hematology/Oncology, Kyungpook National University Hospital, Daegu.
authors.forename: Sang-Kyun
authors.initials: SK
authors.lastname: Sohn
authors.affiliation: Department of Hematology/Oncology, Gyeongsang National University Hospital, Jinju.
authors.forename: Hoon-Gu
authors.initials: HG
authors.lastname: Kim
authors.affiliation: Department of Hematology/Oncology, Gyeongsang National University Hospital, Jinju.
authors.forename: Gyeong-Won
authors.initials: GW
authors.lastname: Lee
authors.affiliation: Department of Hematology/Oncology, Kosin University College of Medicine, Busan.
authors.forename: Yang-Soo
authors.initials: YS
authors.lastname: Kim
authors.affiliation: Department of Hematology/Oncology, Kosin University College of Medicine, Busan.
authors.forename: Ho-Sup
authors.initials: HS
authors.lastname: Lee
authors.affiliation: Department of Hematology/Oncology, Dongsan Medical Center, Daegu.
authors.forename: Ki-Young
authors.initials: KY
authors.lastname: Kwon
authors.affiliation: Department of Hematology/Oncology, Dong-a University Hospital, Busan.
authors.forename: Sung-Hyun
authors.initials: SH
authors.lastname: Kim
authors.affiliation: Department of Hematology/Oncology, Sungkyunkwan University Samsung Changwon Hospital, Changwon.
authors.forename: Kyung-Tae
authors.initials: KT
authors.lastname: Park
authors.affiliation: Department of Hematology/Oncology, Pusan National University, Busan.
authors.forename: Joo-Seop
authors.initials: JS
authors.lastname: Chung
authors.affiliation: Department of Hemato-Oncology, Inje University Busan Paik Hospital, Busan.
authors.forename: Won-Sik
authors.initials: WS
authors.lastname: Lee
authors.affiliation: Department of Hemato-Oncology, Inje University Busan Paik Hospital, Busan.
authors.forename: Sang-Min
authors.initials: SM
authors.lastname: Lee
authors.affiliation: Department of Hematology/Oncology, Yeungnam University Medical Center, Daegu.
authors.forename: Myung-Soo
authors.initials: MS
authors.lastname: Hyun
authors.affiliation: Division of Hematology, Ulsan University Hospital, Ulsan.
authors.forename: Hawk
authors.initials: H
authors.lastname: Kim
authors.affiliation: Department of Hematology/Oncology, Daegu Catholic University Medical Center, Daegu, Korea.
authors.forename: Hun-Mo
authors.initials: HM
authors.lastname: Ryoo
authors.affiliation: Department of Hematology/Oncology, Daegu Catholic University Medical Center, Daegu, Korea.
authors.forename: Sung-Hwa
authors.initials: SH
authors.lastname: Bae
authors.affiliation: Department of Hemato-Oncology, Inje University Haeundae Paik Hospital, Busan.
authors.forename: Young-Don
authors.initials: YD
authors.lastname: Joo
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000741:Anemia, Aplastic / Q000097:blood / Q000628:therapy; D001565:Benzoates / Q000008:administration & dosage*; D000077588:Deferasirox; D017707:Erythrocyte Transfusion / Q000009:adverse effects*; D005293:Ferritins / Q000097:blood*; D006801:Humans; D007501:Iron / Q000097:blood*; D007502:Iron Chelating Agents / Q000008:administration & dosage*; D019190:Iron Overload / Q000097:blood / Q000188:drug therapy / Q000209:etiology; D015470:Leukemia, Myeloid, Acute / Q000097:blood / Q000628:therapy; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000097:blood / Q000628:therapy; D011446:Prospective Studies; D014230:Triazoles / Q000008:administration & dosage*
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D007501:Iron; D000077588:Deferasirox