Chalmers Anna W, Shammo Jamile M
Department of Internal Medicine, Division of Hematology/Oncology, Rush University Medical Center, Chicago, IL, USA.
Ther Clin Risk Manag. 2016 Feb 15;12:201-8. doi: 10.2147/TCRM.S82449. eCollection 2016.
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.
依赖输血的贫血是多种血液系统疾病的常见特征，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。由于缺乏排泄过量铁的生理机制，长期输血最终会导致铁过载。若不加以纠正，铁过载会导致终末器官损伤，进而引起心脏、肝脏和内分泌功能障碍/衰竭。铁螯合剂可与铁形成复合物，促进铁的清除，从而用于减轻铁过载。在某些依赖输血的贫血（如β地中海贫血）中，铁螯合已被证明可减少器官功能障碍并提高生存率。美国食品药品监督管理局已批准多种螯合剂用于治疗铁过载，包括去铁胺、地拉罗司和去铁酮。选择铁螯合剂时需考虑多种因素，包括给药方案、给药途径、耐受性和副作用。地拉罗司是一种口服铁螯合剂，在多种血液系统疾病中已证实其有效性和安全性。地拉罗司有两种剂型，一种是用于混悬液的片剂，另一种是新型片剂。本文旨在概述铁过载，重点介绍地拉罗司及其最近获批的用于减少血液系统疾病中输血性铁过载的剂型Jadenu(®)。
