新型口服铁螯合剂——地拉罗司的研究进展

地拉罗司是一种新型口服铁螯合剂,它是美国FDA批准的第一个能够常规使用的口服驱铁剂,获准在≥2岁、输血造成的慢性铁负荷过多的患者中使用,在欧洲它被推荐作为6岁以上地中海贫血铁过载患者的一线用药,国内目前正在进行临床研究。本文主要就近年来有关地拉罗司的相关研究及最新进展进行介绍。Ⅱ、Ⅲ期临床试验及药代动力学研究均表明,地拉罗司具有良好的安全性和耐受性,且显著降低心脏、肝脏铁负荷,易于被患者接受。地拉罗司常见不良反应为胃肠道症状及皮疹,但近年来报道了一些罕见不良反应事件,应引起我们的重视,尤其是针对特殊人群应谨慎用药。地拉罗司治疗适应证有所放宽,包括接受auto-SCT治疗的患者以及由Fanconi综合症所致的可逆性肾功能不全的患者。地拉罗司常用剂量并非绝对适用于每个患者,临床医师应根据实际病情及相关指标调整用药剂量;铁蛋白并不是一项唯一且可靠的监测铁螯合剂疗效及调整DFS剂量的指标。另外,本文还介绍了地拉罗司的一些新特性,例如抗真菌、抗细胞增殖等作用。

Deferasirox——A New Oral Iron Chelator-Review

Deferasirox is a new oral iron chelator.It is the first oral iron chelator approved in USA by FDA for transfusion-dependent patients above 2 years suffering from severe chronic iron overload.It is also recommended as the initial therapy for patients over the age of 6 years who are suffering from beta-thalassaemia.The clinical study is developing in China.This review focuses the related studies and the latest progression about deferasirox.The phase Ⅱ and Ⅲ clinical trials and pharmacokinetics indicated that deferasirox is a safety and effective oral iron chelator,can significantly decrease the myocardial and hepatic iron load,also is easy to accept for patients.The common adverse reactions are gastrointestinal symptom and rash.But it was recently reported that deferasirox has some rare adverse events to which we must attach importance,especially for the special people.Besides the patients with chronic iron overload resulting from blood transfusions（transfusional hemosiderosis）,the drug is also used for the patients who has accepted auto-SCT or suffered from reversible renal inadequacy caused by Fanconi syndrome.The standard dosage is not useful to every patient.The clinician should adjust dosage based on the patient′s condition and related indexes.The serum ferritin is not one and reliable index to monitoring the effect and adjust the dosage.Otherwise,this review recommends some new characters of deferasirox,e.g.anti-fungus,anti-cell proliferation and so on.