Moukalled Nour M, Bou-Fakhredin Rayan, Taher Ali T
Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon.
Mediterr J Hematol Infect Dis. 2018 Nov 1;10(1):e2018066. doi: 10.4084/MJHID.2018.066. eCollection 2018.
Thalassemia incorporates a broad clinical spectrum characterized by decreased or absent production of normal hemoglobin leading to decreased red blood cell survival and ineffective erythropoiesis. Chronic iron overload remains an inevitable complication resulting from regular blood transfusions (transfusion-dependent) and/or increased iron absorption (mainly non-transfusion-dependent thalassemia), requiring adequate treatment to prevent the significant associated morbidity and mortality. Iron chelation therapy has become a cornerstone in the management of thalassemia patients, leading to improvements in their outcome and quality of life. Deferasirox (DFX), an oral iron chelating agent, is approved for use in transfusion dependent and non-transfusion-dependent thalassemia and has shown excellent efficacy in this setting. We herein present an updated review of the role of deferasirox in thalassemia, exploring over a decade of experience, which has documented its effectiveness and convenience; in addition to its manageable safety profile.
地中海贫血具有广泛的临床谱,其特征为正常血红蛋白生成减少或缺乏,导致红细胞存活期缩短和无效造血。慢性铁过载仍然是定期输血(输血依赖型)和/或铁吸收增加(主要是非输血依赖型地中海贫血)导致的不可避免的并发症,需要进行充分治疗以预防显著的相关发病率和死亡率。铁螯合疗法已成为地中海贫血患者管理的基石,可改善其预后和生活质量。地拉罗司(DFX)是一种口服铁螯合剂,已被批准用于输血依赖型和非输血依赖型地中海贫血,并在此情况下显示出优异的疗效。我们在此对德地拉罗司在地中海贫血中的作用进行最新综述,探讨十多年来的经验,这些经验证明了其有效性、便利性以及可控的安全性。