title: [Efficacy and safety of deferasirox in aplastic anemia patients with iron overload: a single arm, multi-center,prospective study in China].
abstract: OBJECTIVE
  To explore the efficacy and safety of deferasirox in aplastic anemia (AA)patients with iron overload.
  
  
  METHODS
  A single arm, multi- center, prospective, open- label study was conducted to evaluate absolute change in serum ferritin (SF)from baseline to 12 months of deferasirox administration, initially at a dose of 20 mg·kg(-1)·d(-1), and the safety in 64 AA patients with iron overload.
  
  
  RESULTS
  All patients started their deferasirox treatment with a daily dose of 20 mg · kg(-1) ·d(-1). The mean actual dose was (18.6±3.60) mg · kg(-1)·d(-1). The median SF decreased from 4 924 (2 718- 6 765)μg/L at baseline (n=64) to 3 036 (1 474- 5 551)μg/L at 12 months (n=23) with the percentage change from baseline as 38%. A median SF decrease of 651 (126-2 125)μg/L was observed at the end of study in 23 patients who completed 12 months' treatment, the median SF level decreased by 1 167(580-4 806)μg/L [5 271(3 420-8 278)μg/L at baseline; 3 036(1 474-5 551)μg/L after 12 months' treatment; the percentage change from baseline as 42% ] after 12 months of deferasirox treatment. The most common adverse events (AEs) were increased serum creatinine levels (40.98%), gastrointestinal discomfort (40.98%), elevated liver transaminase (ALT: 21.31%; AST: 13.11%)and proteinuria (24.59%). The increased serum creatinine levels were reversible and non-progressive. Of 38 patients with concomitant cyclosporine use, 12(31.8%)patients had two consecutive values >ULN, 10(26.3%)patients had two consecutive values >1.33 baseline values, but only 1(2.6%)patient's serum creatinine increased more than 1.33 baseline values and exceeded ULN. For both AST and ALT, no patients experienced two post- baseline values >5 ×ULN or >10 × ULN during the whole study. In AA patients with low baseline PLT count (less than 50 × 10(9)/L), there was no decrease for median PLT level during 12 months' treatment period.
  
  
  CONCLUSIONS
  AA patients with iron overload could achieve satisfactory efficacy of iron chelation by deferasirox treatment. The drug was well tolerated with a clinically manageable safety profile and no major adverse events.
authors.affiliation: Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, Tianjin 300020, China.
authors.forename: Jun
authors.initials: J
authors.lastname: Shi
authors.affiliation: Department of Hematology and Research Laboratory of Hematology, West China Hospital of Sichuan University, Chengdu 610041, China.
authors.forename: Hong
authors.initials: H
authors.lastname: Chang
authors.forename: Li
authors.initials: L
authors.lastname: Zhang
authors.forename: Yinqi
authors.initials: Y
authors.lastname: Shao
authors.forename: Neng
authors.initials: N
authors.lastname: Nie
authors.forename: Jing
authors.initials: J
authors.lastname: Zhang
authors.forename: Jinbo
authors.initials: J
authors.lastname: Huang
authors.forename: Li
authors.initials: L
authors.lastname: Zhang
authors.forename: Xudong
authors.initials: X
authors.lastname: Tang
authors.forename: Richeng
authors.initials: R
authors.lastname: Quan
authors.forename: Chunmei
authors.initials: C
authors.lastname: Zheng
authors.forename: Haiyan
authors.initials: H
authors.lastname: Xiao
authors.forename: Dengming
authors.initials: D
authors.lastname: Hu
authors.forename: Lingyan
authors.initials: L
authors.lastname: Hu
authors.forename: Feng
authors.initials: F
authors.lastname: Liu
authors.forename: Yongming
authors.initials: Y
authors.lastname: Zhou
authors.forename: Yizhou
authors.initials: Y
authors.lastname: Zheng
authors.affiliation: Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, Tianjin 300020, China.
authors.forename: Fengkui
authors.initials: F
authors.lastname: Zhang
mesh_terms: D000741:Anemia, Aplastic / Q000188:drug therapy*; D001565:Benzoates / Q000627:therapeutic use*; D001803:Blood Transfusion; D002681:China; D000077588:Deferasirox; D005293:Ferritins / Q000097:blood; D006801:Humans; D007501:Iron / Q000097:blood; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D019190:Iron Overload / Q000188:drug therapy*; D008099:Liver; D011446:Prospective Studies; D014230:Triazoles / Q000627:therapeutic use*
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D007501:Iron; D000077588:Deferasirox