Deferasirox: appraisal of safety and efficacy in long-term therapy

被引:19
作者
Chaudhary, Preeti [1 ]
Pullarkat, Vinod [1 ]
机构
[1] Univ Southern Calif, Keck Sch Med, Jane Ann Nohl Div Hematol, 1441 Eastlake Ave,MC 9172,NOR 3461, Los Angeles, CA 90033 USA
来源
JOURNAL OF BLOOD MEDICINE | 2013年 / 4卷
关键词
deferasirox; iron overload; thalassemia; sickle-cell disease; myelodysplastic syndrome;
D O I
10.2147/JBM.S35478
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Deferasirox is a once-daily, oral iron chelator that is widely used in the management of patients with transfusional hemosiderosis. Several Phase II trials along with their respective extension studies as well as a Phase III trial have established the efficacy and safety of this novel agent in transfusion- dependent patients with beta-thalassemia, sickle-cell disease and bone marrow-failure syndromes, including myelodysplastic syndrome and aplastic anemia. Data from various clinical trials show that a deferasirox dose of 20 mg/kg/day stabilizes serum ferritin levels and liver iron concentration, while a dose of 30-40 mg/kg/day reduces these parameters and achieves negative iron balance in red cell transfusion- dependent patients with iron overload. Across various pivotal clinical trials, deferasirox was well tolerated, with the most common adverse events being gastrointestinal disturbances, skin rash, nonprogressive increases in serum creatinine, and elevations in liver enzyme levels. Longer-term extension studies have also confirmed the efficacy and safety of deferasirox. However, it is essential that patients on deferasirox therapy are monitored regularly to ensure timely management for any adverse events that may occur with long-term therapy.
引用
收藏
页码:101 / 109
页数:9
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