去铁酮治疗输血依赖型地中海贫血患者的安全性和有效性。
Safety and efficacy of iron chelation therapy with deferiprone in patients with transfusion-dependent thalassemia.
机构信息
Division of Genetics, 300 Longwood Avenue, Boston Children's Hospital, Boston, MA 02115, USA.
出版信息
Ther Adv Hematol. 2012 Oct;3(5):299-307. doi: 10.1177/2040620712450252.
Abstract
Deferiprone is an orally active iron-chelating agent used in the management of transfusion-related hemosiderosis. It has been in clinical use for over 20 years and has been shown to be effective in reducing cardiac iron load and improving cardiac function. As cardiac siderosis is the leading cause of death in patients with transfusion-dependent thalassemia, deferiprone helps to improve the overall prognosis of these patients. It is relatively well tolerated with gastrointestinal symptoms being the commonest side effects. Agranulocytosis (0.5%), neutropenia (9%), thrombocytopenia (up to 45%) and arthropathy (20%) are the most important side effects and may require discontinuation of therapy. Regular monitoring of blood counts is recommended for patients on deferiprone therapy.
摘要
地拉罗司是一种口服活性铁螯合剂，用于治疗输血相关的血色素沉着症。它已经在临床上使用了 20 多年，已被证明可有效降低心脏铁负荷并改善心脏功能。由于心脏铁沉积是输血依赖型地中海贫血患者死亡的主要原因，地拉罗司有助于改善这些患者的整体预后。它具有较好的耐受性，最常见的副作用是胃肠道症状。粒细胞缺乏症（0.5%）、中性粒细胞减少症（9%）、血小板减少症（高达 45%）和关节病（20%）是最重要的副作用，可能需要停止治疗。建议接受地拉罗司治疗的患者定期监测血象。
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