PMID- 26876245
OWN - NLM
STAT- MEDLINE
DCOM- 20160706
LR  - 20231111
IS  - 0253-2727 (Print)
IS  - 2707-9740 (Electronic)
IS  - 0253-2727 (Linking)
VI  - 37
IP  - 1
DP  - 2016 Jan
TI  - [Efficacy and safety of deferasirox in aplastic anemia patients with iron 
      overload: a single arm, multi-center,prospective study in China].
PG  - 1-6
LID - 10.3760/cma.j.issn.0253-2727.2016.01.001 [doi]
AB  - 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)mug/L at baseline 
      (n=64) to 3 036 (1 474- 5 551)mug/L at 12 months (n=23) with the percentage change 
      from baseline as 38%. A median SF decrease of 651 (126-2 125)mug/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)mug/L [5 271(3 420-8 278)mug/L at baseline; 3 
      036(1 474-5 551)mug/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 xULN or >10 x 
      ULN during the whole study. In AA patients with low baseline PLT count (less than 
      50 x 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.
FAU - Shi, Jun
AU  - Shi J
AD  - Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, Tianjin 300020, 
      China.
FAU - Chang, Hong
AU  - Chang H
AD  - Department of Hematology and Research Laboratory of Hematology, West China 
      Hospital of Sichuan University, Chengdu 610041, China.
FAU - Zhang, Li
AU  - Zhang L
FAU - Shao, Yinqi
AU  - Shao Y
FAU - Nie, Neng
AU  - Nie N
FAU - Zhang, Jing
AU  - Zhang J
FAU - Huang, Jinbo
AU  - Huang J
FAU - Zhang, Li
AU  - Zhang L
FAU - Tang, Xudong
AU  - Tang X
FAU - Quan, Richeng
AU  - Quan R
FAU - Zheng, Chunmei
AU  - Zheng C
FAU - Xiao, Haiyan
AU  - Xiao H
FAU - Hu, Dengming
AU  - Hu D
FAU - Hu, Lingyan
AU  - Hu L
FAU - Liu, Feng
AU  - Liu F
FAU - Zhou, Yongming
AU  - Zhou Y
FAU - Zheng, Yizhou
AU  - Zheng Y
FAU - Zhang, Fengkui
AU  - Zhang F
AD  - Institute of Hematology and Blood Diseases Hospital, CAMS & PUMC, Tianjin 300020, 
      China.
LA  - chi
SI  - ClinicalTrials.gov/NCT01546415
PT  - Clinical Study
PT  - Journal Article
PT  - Multicenter Study
PL  - China
TA  - Zhonghua Xue Ye Xue Za Zhi
JT  - Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi
JID - 8212398
RN  - 0 (Benzoates)
RN  - 0 (Iron Chelating Agents)
RN  - 0 (Triazoles)
RN  - 9007-73-2 (Ferritins)
RN  - E1UOL152H7 (Iron)
RN  - V8G4MOF2V9 (Deferasirox)
SB  - IM
MH  - Anemia, Aplastic/*drug therapy
MH  - Benzoates/*therapeutic use
MH  - Blood Transfusion
MH  - China
MH  - Deferasirox
MH  - Ferritins/blood
MH  - Humans
MH  - Iron/blood
MH  - Iron Chelating Agents/*therapeutic use
MH  - Iron Overload/*drug therapy
MH  - Liver
MH  - Prospective Studies
MH  - Triazoles/*therapeutic use
PMC - PMC7342302
EDAT- 2016/02/16 06:00
MHDA- 2016/07/07 06:00
PMCR- 2016/01/01
CRDT- 2016/02/16 06:00
PHST- 2016/02/16 06:00 [entrez]
PHST- 2016/02/16 06:00 [pubmed]
PHST- 2016/07/07 06:00 [medline]
PHST- 2016/01/01 00:00 [pmc-release]
AID - cjh-37-01-001 [pii]
AID - 10.3760/cma.j.issn.0253-2727.2016.01.001 [doi]
PST - ppublish
SO  - Zhonghua Xue Ye Xue Za Zhi. 2016 Jan;37(1):1-6. doi: 
      10.3760/cma.j.issn.0253-2727.2016.01.001.