去铁酮与去铁胺对重型地中海贫血患者生存及心脏疾病的比较影响：一项回顾性分析

Comparative effects of deferiprone and deferoxamine on survival and cardiac disease in patients with thalassemia major: a retrospective analysis.

作者信息

Piga Antoni, Gaglioti Carmen, Fogliacco Eugenia, Tricta Fernando

机构信息

Department of Pediatric Hematology, University of Turin, Italy.

出版信息

Haematologica. 2003 May;88(5):489-96.

PMID:12745268

Abstract

BACKGROUND AND OBJECTIVES

Iron-induced cardiac disease remains the main cause of death in patients with thalassemia major, despite chelation therapy with deferoxamine. Deferiprone is an iron chelator that has the potential to be more effective than deferoxamine in removing intracellular iron from the heart. However, to date, no study has been designed to examine the frequency of cardiac complications and survival as the primary outcomes of a comparative study between these two chelators. This retrospective study assessed the survival and the occurrence of cardiac disease in all patients with thalassemia major treated for at least 4 years with deferiprone or deferoxamine at a single center.

DESIGN AND METHODS

The patients were, on average, 18.4 years old at the start of the review period and were followed up, on average, for 6 years. At baseline there was no significant difference in the percentage of patients with cardiac disease in the two therapy groups.

RESULTS

At the end of the study, cardiac dysfunction, expressed as worsening of pre-existing cardiac abnormality or development of new cardiac disease, was diagnosed in 2 (4%) of the 54 deferiprone-treated patients and in 15 (20%) of the 75 deferoxamine-treated patients, from the first to the last measurement (p = 0.007). The Kaplan Meier analysis of cardiac disease-free survival over the 5-year period was significantly more favorable in the deferiprone group (p = 0.003).

INTERPRETATION AND CONCLUSIONS

None of the patients treated with deferiprone died, while 3 of the patients treated with deferoxamine died because of irreversible worsening of their cardiac condition during the study period. Findings from this study suggest that long-term therapy with deferiprone provides a greater cardio-protective effect against the toxicity of iron overload than does subcutaneous deferoxamine. Formal prospective studies are warranted to confirm this effect.

摘要

背景与目的

尽管采用去铁胺进行螯合治疗，但铁诱导的心脏疾病仍是重型地中海贫血患者的主要死因。地拉罗司是一种铁螯合剂，在清除心脏细胞内铁方面可能比去铁胺更有效。然而，迄今为止，尚无研究将心脏并发症的发生率和生存率作为这两种螯合剂对比研究的主要结局指标。这项回顾性研究评估了在单一中心接受地拉罗司或去铁胺治疗至少4年的所有重型地中海贫血患者的生存情况及心脏疾病的发生情况。

设计与方法

在回顾期开始时，患者平均年龄为18.4岁，平均随访6年。基线时，两个治疗组中心脏疾病患者的百分比无显著差异。

结果

在研究结束时，从首次测量到最后一次测量，54例接受地拉罗司治疗的患者中有2例（4%）被诊断为心脏功能障碍，表现为原有心脏异常加重或出现新的心脏疾病；75例接受去铁胺治疗的患者中有15例（20%）出现心脏功能障碍（p = 0.007）。地拉罗司组5年无心脏病生存率的Kaplan Meier分析结果明显更优（p = 0.003）。