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铁螯合剂方案对重型地中海贫血患者心脏和肝脏铁含量的降低效果:一项临床观察性研究。

The efficacy of iron chelator regimes in reducing cardiac and hepatic iron in patients with thalassaemia major: a clinical observational study.

作者信息

Berdoukas Vasilios, Chouliaras Giorgos, Moraitis Panagiotis, Zannikos Kirykos, Berdoussi Eleni, Ladis Vassilios

机构信息

Thalassaemia Unit, First Department of Paediatrics, University of Athens, Aghia Sophia"Children's Hospital, Athens, Greece.

出版信息

J Cardiovasc Magn Reson. 2009 Jun 28;11(1):20. doi: 10.1186/1532-429X-11-20.

DOI:10.1186/1532-429X-11-20
PMID:19558722
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC2713224/
Abstract

BACKGROUND

Available iron chelation regimes in thalassaemia may achieve different changes in cardiac and hepatic iron as assessed by MR. The aim of this study was to assess the efficacy of four available iron chelator regimes in 232 thalassaemia major patients by assessing the rate of change in repeated measurements of cardiac and hepatic MR.

RESULTS

For the heart, deferiprone and the combination of deferiprone and deferoxamine significantly reduced cardiac iron at all levels of iron loading. As patients were on deferasirox for a shorter time, a second analysis ("Initial interval analysis") assessing the change between the first two recorded MR results for both cardiac and hepatic iron (minimum interval 12 months) was made. Combination therapy achieved the most rapid fall in cardiac iron load at all levels and deferiprone alone was significantly effective with moderate and mild iron load. In the liver, deferasirox effected significant falls in iron load and combination therapy resulted in the most rapid decline.

CONCLUSION

With the knowledge of the efficacy of the different available regimes and the specific iron load in the heart and the liver, appropriate tailoring of chelation therapy should allow clearance of iron. Combination therapy is best in reducing both cardiac and hepatic iron, while monotherapy with deferiprone or deferasirox are effective in the heart and liver respectively. The outcomes of this study may be useful to physicians as to the chelation they should prescribe according to the levels of iron load found in the heart and liver by MR.

摘要

背景

地中海贫血中现有的铁螯合方案通过磁共振成像(MR)评估可能会使心脏和肝脏铁含量发生不同变化。本研究的目的是通过评估心脏和肝脏MR重复测量的变化率,来评估四种现有的铁螯合剂方案对232例重型地中海贫血患者的疗效。

结果

对于心脏,去铁酮以及去铁酮与去铁胺联合使用在所有铁负荷水平下均能显著降低心脏铁含量。由于患者使用地拉罗司的时间较短,因此进行了第二项分析(“初始间隔分析”),评估心脏和肝脏铁的前两次记录的MR结果之间的变化(最短间隔12个月)。联合治疗在所有水平下使心脏铁负荷下降最快,单独使用去铁酮对中度和轻度铁负荷也有显著效果。在肝脏方面,地拉罗司使铁负荷显著下降,联合治疗导致下降最快。

结论

了解不同现有方案的疗效以及心脏和肝脏中的特定铁负荷后,适当调整螯合疗法应能清除铁。联合治疗在降低心脏和肝脏铁含量方面效果最佳,而去铁酮或地拉罗司单药治疗分别对心脏和肝脏有效。本研究结果可能有助于医生根据MR检测到的心脏和肝脏铁负荷水平来选择螯合疗法。

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