IRON-CHELATION THERAPY WITH ORAL DEFERIPRONE IN PATIENTS WITH THALASSEMIA MAJOR
IRON-CHELATION THERAPY WITH ORAL DEFERIPRONE IN PATIENTS WITH THALASSEMIA MAJOR
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DOI：
10.1056/nejm199504063321404
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发表时间：
1995-04-06
影响因子：
158.5
通讯作者：
KOREN, G
中科院分区：
文献类型：
--
作者：
OLIVIERI, NF;BRITTENHAM, GM;KOREN, G
背景。为了确定口服活性铁螯合剂去铁酮（1,2-二甲基-3-羟基吡啶-4-酮）是否能有效治疗重型地中海贫血患者的铁超负荷，我们对 21 名无法或不愿意使用肠外去铁胺标准螯合疗法的患者进行了一项去铁酮前瞻性试验。方法。每年通过肝活检标本的化学分析或磁化率测量来确定肝铁储存。使用详细的临床和实验室研究来监测安全性和依从性。结果。患者接受去铁酮治疗的平均时间 (+/-SE) 为 3.1+/-0.3 年。先前使用去铁胺螯合疗法无效的 10 名患者的初始肝铁浓度至少为每克肝脏 80 μmol，湿重 - 与铁超载并发症相关的值。所有 10 名患者的肝铁浓度均下降，从 125.3+/-11.5 降至 60.3+/-9.6 μmol/g（P
Background. To determine whether the orally active iron chelator deferiprone (1,2-dimethyl-3-hydroxypyridin-4-one) is efficacious in the treatment of iron overload in patients with thalassemia major, we conducted a prospective trial of deferiprone in 21 patients unable or unwilling to use standard chelation therapy with parenteral deferoxamine.Methods. Hepatic iron stores were determined yearly by chemical analysis of liver-biopsy specimens or magnetic-susceptibility measurements. Detailed clinical and laboratory studies were used to monitor safety and compliance.Results. The patients received deferiprone therapy for a mean (+/-SE) of 3.1+/-0.3 years. Ten patients in whom previous chelation therapy with deferoxamine had been ineffective had initial hepatic iron concentrations of at least 80 mu mol per gram of liver, wet weight - values associated with complications of iron overload. Hepatic iron concentrations decreased in all 10 patients, from 125.3+/-11.5 to 60.3+/-9.6 mu mol per gram (P