Belmont Ami, Kwiatkowski Janet L
a Alpert Medical School of Brown University , Providence , RI , USA.
b Children's Hospital of Philadelphia, Division of Hematology and Perelman School of Medicine at the University of Pennsylvania, Department of Pediatrics , Philadelphia , PA , USA.
Expert Rev Hematol. 2017 Jun;10(6):493-503. doi: 10.1080/17474086.2017.1318052.
Transfusional iron overload can lead to hepatic fibrosis, arrhythmias and congestive heart failure and a number of endocrinopathies. Deferiprone is an oral iron chelator approved for use in the United States as a second line agent for the treatment of transfusional iron overload in patients with thalassemia. Areas covered: This article will review the data regarding the efficacy of deferiprone for iron chelation and prevention and reversal of iron related complications, the drug's adverse effect profile, and the use of this drug in combination regimens. Expert commentary: Extensive data support that deferiprone is particularly efficacious at cardiac iron removal and therefore, a chelator regimen that contains deferiprone is generally recommended when there is significant cardiac iron loading and/or in the setting of iron-related cardiac disease. The most concerning side effects of deferiprone are agranulocytosis and milder forms of neutropenia, which require appropriate monitoring and patient/provider education.
输血性铁过载可导致肝纤维化、心律失常、充血性心力衰竭以及多种内分泌疾病。去铁酮是一种口服铁螯合剂，在美国被批准作为治疗地中海贫血患者输血性铁过载的二线药物。涵盖领域：本文将综述有关去铁酮在铁螯合以及预防和逆转铁相关并发症方面的疗效数据、该药物的不良反应情况以及该药物在联合治疗方案中的应用。专家评论：大量数据支持去铁酮在清除心脏铁方面特别有效，因此，当存在显著的心脏铁负荷和/或在铁相关心脏病的情况下，通常推荐包含去铁酮的螯合治疗方案。去铁酮最令人担忧的副作用是粒细胞缺乏症和较轻形式的中性粒细胞减少症，这需要进行适当的监测以及对患者/医疗服务提供者进行教育。
