Evaluation of a new tablet formulation of deferasirox to reduce chronic iron overload after long-term blood transfusions.
Evaluation of a new tablet formulation of deferasirox to reduce chronic iron overload after long-term blood transfusions.
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DOI：
10.2147/tcrm.s82449
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发表时间：
2016
影响因子：
2.8
通讯作者：
Shammo JM
中科院分区：
文献类型：
--
作者：
Chalmers AW;Shammo JM
输血依赖性贫血是一系列血液病的共同特征，包括地中海贫血、镰状细胞病、再生障碍性贫血、骨髓纤维化和骨髓增生异常综合征。在缺乏排泄过量铁的生理机制的情况下，慢性输血最终会导致铁超载。如果不纠正，铁超载可能会导致终末器官损伤，导致心脏、肝脏和内分泌功能障碍/衰竭。铁络合剂被用来减少铁的过载，因为它们与铁形成络合物，导致其清除。铁络合物已被证明可以减少某些输血依赖型贫血(如β-地中海贫血)的器官功能障碍并提高存活率。几种螯合剂已被美国食品和药物管理局批准用于治疗铁超载，包括去铁胺、去铁酮和去铁西罗克斯。在选择铁络合剂时，必须考虑各种因素，包括给药计划、给药途径、耐受性和副作用概况。Deferasirox是一种口服铁络合剂，已被证明对多种血液系统疾病有效且安全。去铁西洛有两种剂型，一种是悬浮片，另一种是新的片剂形式。这篇文章旨在提供铁超载的概述，重点是deferasirox，以及其最近批准的用于降低血液系统疾病输血铁超载的配方Jadeu®。
Transfusion-dependent anemia is a common feature in a wide array of hematological disorders, including thalassemia, sickle cell disease, aplastic anemia, myelofibrosis, and myelo-dysplastic syndromes. In the absence of a physiological mechanism to excrete excess iron, chronic transfusions ultimately cause iron overload. Without correction, iron overload can lead to end-organ damage, resulting in cardiac, hepatic, and endocrine dysfunction/failure. Iron chelating agents are utilized to reduce iron overload, as they form a complex with iron, leading to its clearance. Iron chelation has been proven to decrease organ dysfunction and improve survival in certain transfusion-dependent anemias, such as β-thalassemia. Several chelating agents have been approved by the United States Food and Drug Administration for the treatment of iron overload, including deferoxamine, deferiprone, and deferasirox. A variety of factors have to be considered when choosing an iron chelator, including dosing schedule, route of administration, tolerability, and side effect profile. Deferasirox is an orally administered iron chelator with proven efficacy and safety in multiple hematological disorders. There are two formulations of deferasirox, a tablet for suspension, and a new tablet form. This paper is intended to provide an overview of iron overload, with a focus on deferasirox, and its recently approved formulation Jadenu® for the reduction of transfusional iron overload in hematological disorders.