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地拉罗司的临床应用:实际患者管理

Clinical application of deferasirox: practical patient management.

作者信息

Vichinsky Elliott

机构信息

Hematology/Oncology Department, Children's Hospital and Research Center at Oakland, Oakland, California 94609, USA.

出版信息

Am J Hematol. 2008 May;83(5):398-402. doi: 10.1002/ajh.21119.

Abstract

Deferasirox (Exjade, ICL670) is a once-daily, oral iron chelation agent that is now widely available for the treatment of transfusional hemosiderosis in adult and pediatric patients aged > or =2 years of age. Clinical evaluation has established the efficacy and safety of this novel agent in patients with a variety of chronic anemias. Deferasirox represents a significant advance in the treatment of iron overload, as the availability of an effective oral therapy has the potential to relieve many patients from the burden of frequent parenteral therapy with the previous reference standard iron chelator, deferoxamine. The most common drug-related adverse events seen in the core registration trials were gastrointestinal disturbances, rash, mild and nonprogressive increases in serum creatinine levels, and elevations in liver enzyme levels. Most events were transient, mild-to-moderate in severity, and easily managed without discontinuation of treatment. As with any new agent, it is important that treating physicians are familiar with the adverse event profile of deferasirox and how the associated effects can be readily managed to ensure optimal use of this important treatment.

摘要

地拉罗司(恩瑞格,ICL670)是一种每日服用一次的口服铁螯合剂,目前已广泛用于治疗年龄≥2岁的成人和儿童患者的输血性含铁血黄素沉着症。临床评估已证实这种新型药物在各类慢性贫血患者中的疗效和安全性。地拉罗司代表了铁过载治疗方面的一项重大进展,因为有效的口服疗法的出现有可能使许多患者摆脱以往参照标准铁螯合剂去铁胺频繁进行肠胃外治疗的负担。在核心注册试验中观察到的最常见的与药物相关的不良事件是胃肠道不适、皮疹、血清肌酐水平轻度且非进行性升高以及肝酶水平升高。大多数事件是短暂的,严重程度为轻度至中度,并且在不中断治疗的情况下易于处理。与任何新药一样,治疗医生熟悉地拉罗司的不良事件谱以及如何轻松处理相关效应以确保最佳使用这种重要治疗方法非常重要。

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