Randomized controlled trial of deferiprone or deferoxamine in beta-thalassemia major patients with asymptomatic myocardial siderosis
Randomized controlled trial of deferiprone or deferoxamine in beta-thalassemia major patients with asymptomatic myocardial siderosis
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DOI：
10.1182/blood-2005-07-2948
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发表时间：
2006-05-01
期刊：
影响因子：
20.3
通讯作者：
Galanello, R
中科院分区：
文献类型：
--
作者：
Pennell, DJ;Berdoukas, V;Galanello, R
大多数乙型地中海贫血的死亡主要是由于铁超载引起的心脏并发症。不同的螯合剂对心肌铁沉着的影响可能存在差异。一项随机对照试验在61例先前维持皮下去铁胺治疗的患者中进行。主要终点是维持皮下去铁胺治疗或转向口服去铁胺单药治疗的患者1年内心肌铁沉着（心肌T2*）的变化。去铁酮剂量为92 mg/kg/d，去铁胺剂量为43 mg/kg，疗程为5.7 d/周。依从性分别为94% +/- 5.3%和93% +/- 9.7% （P = 0.81）。去铁胺组对心肌T2*的改善明显大于去铁胺组（27% vs 13%; P = 0.023）。去替酚酮组左室射血分数明显升高（3.1% vs 0.3%绝对单位；P = 0.003）。肝铁水平（-0.93 mg/g干重vs -1.54 mg/g干重，P = 0.40）和血清铁蛋白水平（-181 μ g/L vs -466 μ g/L, P = 0.16）各组间差异不显著。最常见的不良事件是接受去铁酚酮治疗的患者的短暂胃肠道症状和去铁胺输注部位的局部反应。无粒细胞缺乏症发作。在改善重度β -地中海贫血患者无症状心肌铁沉着方面，1年内铁易感性单药治疗明显比去铁胺更有效。
Most deaths in beta-thalassemia major result from cardiac complications due to iron overload. Differential effects on myocardial siderosis may exist between different chelators. A randomized controlled trial was performed in 61 patients previously maintained on subcutaneous deferoxamine. The primary end point was the change in myocardial siderosis (myocardial T2*) over 1 year in patients maintained on subcutaneous deferoxamine or those switched to oral deferiprone monotherapy. The dose of deferiprone was 92 mg/kg/d and deferoxamine was 43 mg/kg for 5.7 d/wk. Compliance was 94% +/- 5.3% and 93% +/- 9.7% (P = .81), respectively. The improvement in myocardial T2* was significantly greater for deferiprone than deferoxamine (27% vs 13%; P = .023). Left ventricular ejection fraction increased significantly more in the deferiprone-treated group (3.1% vs 0.3% absolute units; P = .003). The changes in liver iron level (-0.93 mg/g dry weight vs -1.54 mg/g dry weight; P = .40) and serum ferritin level (-181 mu g/L vs -466 mu g/L; P = .16), respectively, were not significantly different between groups. The most frequent adverse events were transient gastrointestinal symptoms for deferiprone-treated patients and local reactions at the infusion site for deferoxamine. There were no episodes of agranulocytosis. Deferiprone monotherapy was significantly more effective than deferoxamine over 1 year in improving asymptomatic myocardial siderosis in beta-thalassemia major.