SAFETY AND EFFICACY OF 4 YEARS OF DEFERASIROX TREATMENT FOR SICKLE CELL DISEASE PATIENTS

被引：3
作者：
Vlachaki, Efthymia [1 ]
Mainou, Maria [2 ]
Bekiari, Eleni [2 ]
Vetsiou, Evaggelia [1 ]
Tsapas, Apostolos [2 ]
机构：
[1] Aristotle Univ Thessaloniki, Hippokratio Gen Hosp, Thalassemia Unit, Dept Med 2, Thessaloniki 54642, Greece
[2] Aristotle Univ Thessaloniki, Metab Dis Unit, Dept Med 2, Thessaloniki 54642, Greece
来源：
HEMOGLOBIN | 2013年 / 37卷 / 01期
关键词：
Deferasirox (DFRA); Chelation treatment; Sickle cell disease; ORAL IRON CHELATOR; THERAPY; DEFERIPRONE; OVERLOAD; THALASSEMIA; MORBIDITY; MORTALITY;
D O I：
10.3109/03630269.2012.746696
中图分类号：
Q5 [生物化学]; Q7 [分子生物学];
学科分类号：
071010 ; 081704 ;
摘要：
Deferasirox (DFRA) is a novel oral chelator agent for treatment of iron overload. Although well established in the treatment of beta-thalassemia major (beta-TM), it has not yet been fully investigated in patients with sickle cell disease. The aim of this report is to present the preliminary results of a pilot study assessing the effect of 4 years of DFRA treatment in six patients with sickle cell disease who are in need of recurrent transfusions. Our results show a significant reduction of ferritin levels and improvement of liver hemosiderosis, assessed by means of magnetic resonance imaging T2* (MRI T2*). None of the patients presented any serious adverse effects and the treatment was well tolerated. These results are in accordance with previous studies about the use of DFRA in sickle cell disease.
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页码：94 / 100
页数：7
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