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Combined chelation with high-dose deferiprone and deferoxamine to improve survival and restore cardiac function effectively in patients with transfusion-dependent thalassemia presenting severe cardiac complications.

联合大剂量去铁酮和去铁胺螯合治疗出现严重心脏并发症的输血依赖性地中海贫血患者,有效改善生存率和恢复心功能。

  • 影响因子:1.94
  • DOI:10.1007/s00277-020-04196-y
  • 作者列表:"Chuang TY","Li JP","Weng TF","Wu KH","Chao YH
  • 发表时间:2020-10-01
Abstract

:Iron overload-induced cardiomyopathy is the leading cause of death in patients with transfusion-dependent thalassemia (TDT). The mortality is extremely high in these patients with severe cardiac complications, and how to rescue them remains a challenge. It is reasonable to use combined chelation with deferiprone (L1) and deferoxamine (DFO) because of their shuttle and synergistic effects on iron chelation. Here, seven consecutive patients with TDT who had severe cardiac complications between 2002 and 2019 and received combined chelation therapy with oral high-dose L1 (100 mg/kg/day) and continuous 24-h DFO infusion (50 mg/kg/day) in our hospital were reported. Survival for eight consecutive patients receiving DFO monotherapy for their severe cardiac complications between 1984 and 2001 was compared. We found that combined chelation therapy with high-dose L1 and DFO was efficient to improve survival and cardiac function in patients with TDT presenting severe cardiac complications. Reversal of arrhythmia to sinus rhythm was noted in all patients. Their 1-month follow-up left ventricular ejection fraction increased significantly (P < 0.001). There were no deaths, and all patients were discharged from hospital with good quality of life. In contrast, all the eight patients receiving DFO monotherapy died (P < 0.001). Accordingly, combined chelation therapy with high-dose L1 and DFO should be considered in patients with TDT presenting cardiac complications.

摘要

: 铁过载诱导的心肌病是输血依赖性地中海贫血 (TDT) 患者死亡的主要原因。这些严重心脏并发症患者的死亡率极高,如何抢救仍然是一个挑战。使用去铁酮 (L1) 和去铁胺 (DFO) 的组合螯合是合理的,因为它们对铁螯合具有穿梭和协同作用。在这里,我们报告了7例连续的TDT患者,他们在2002年至2019年间出现严重的心脏并发症,并在我们医院接受了口服大剂量L1 (100 mg/kg/天) 和连续24小时DFO输注 (50 mg/kg/天) 的联合螯合治疗。比较了1984年至2001年间连续8例接受DFO单药治疗的严重心脏并发症患者的生存率。我们发现大剂量L1和DFO联合螯合治疗可有效改善存在严重心脏并发症的TDT患者的生存率和心功能。所有患者心律失常均逆转为窦性心律。随访1个月左室射血分数明显升高 (p <0.001)。无死亡病例,所有患者均出院,生活质量良好。相比之下,接受DFO单药治疗的所有8例患者死亡 (p <0.001)。因此,在出现心脏并发症的TDT患者中应考虑高剂量L1和DFO的联合螯合治疗。

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