Efficacy and safety of deferasirox in aplastic anemia patients with iron overload: a single arm, multi-center,prospective study in China

• Published in: Zhōnghuá xuèyèxué zázhì 2016-01, Vol.37 (1), p.1-6
• Main Authors: Shi, Jun, Chang, Hong, Zhang, Li, Shao, Yinqi, Nie, Neng, Zhang, Jing, Huang, Jinbo, Tang, Xudong, Quan, Richeng, Zheng, Chunmei, Xiao, Haiyan, Hu, Dengming, Hu, Lingyan, Liu, Feng, Zhou, Yongming, Zheng, Yizhou, Zhang, Fengkui
• Format: Article
• Language: Chinese
• Subjects: Anemia, Aplastic - drug therapy; Benzoates - therapeutic use; Blood Transfusion; China; Ferritins - blood; Humans; Iron - blood; Iron Chelating Agents - therapeutic use; Iron Overload - drug therapy; Liver; Prospective Studies; Triazoles - therapeutic use
• ISSN: 0253-2727
• DOI: 10.3760/cma.j.issn.0253-2727.2016.01.001

To explore the efficacy and safety of deferasirox in aplastic anemia (AA)patients with iron overload. 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. 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 m