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MRI 多中心前瞻性研究:地拉罗司治疗重型地中海贫血患者与去铁酮和去铁胺的对比。

MRI multicentre prospective survey in thalassaemia major patients treated with deferasirox versus deferiprone and desferrioxamine.

机构信息

Magnetic Resonance Imaging Unit, Fondazione G. Monasterio CNR-Regione Toscana, Pisa, Italy.

Unità Operativa Complessa Ematologia con Talassemia, ARNAS Civico "Benfratelli-Di Cristina", Palermo, Italy.

出版信息

Br J Haematol. 2018 Dec;183(5):783-795. doi: 10.1111/bjh.15595. Epub 2018 Oct 18.

Abstract

We prospectively assessed the efficacy of deferasirox versus deferiprone or desferrioxamine as monotherapy in thalassaemia major (TM) patients by magnetic resonance imaging (MRI). We selected the patients enrolled in the Myocardial Iron Overload in Thalassaemia network who received only one chelator between two MRIs (deferasirox = 235, deferiprone = 142, desferrioxamine = 162). Iron overload was measured by T2* technique and biventricular function by cine images. Among the patients with baseline myocardial iron, in all three groups there was a significant improvement in global heart T2* values. The deferiprone and desferrioxamine groups showed a significant improvement in left ventricular ejection fraction (LVEF). Only the deferiprone group showed a significant improvement in right ventricular ejection fraction (RVEF). The improvement in global heart T2* was significantly lower in the deferasirox versus the deferiprone group. The improvement in the LVEF was significantly higher in the deferiprone and desferrioxamine groups than in the deferasirox group and the improvement in the RVEF was significantly higher in the deferiprone than in deferasirox group. Among the patients with baseline hepatic iron, the changes in hepatic iron were comparable in deferasirox versus the other groups. Deferasirox monotherapy was less effective than deferiprone in improving myocardial siderosis and biventricular function and less effective than desferrioxamine in improving the LVEF.

摘要

我们前瞻性地评估了去铁酮、地拉罗司或去铁胺作为单一疗法在重型地中海贫血(TM)患者中的疗效,方法是通过磁共振成像(MRI)。我们选择了在 Myocardial Iron Overload in Thalassaemia 网络中入组的患者,这些患者在两次 MRI 之间仅接受一种螯合剂(去铁酮=235,地拉罗司=142,去铁胺=162)。铁超负荷通过 T2技术和电影图像测量心功能。在有基线心肌铁的患者中,三组患者的全心 T2值均显著改善。地拉罗司和去铁胺组的左心室射血分数(LVEF)显著改善。只有地拉罗司组的右心室射血分数(RVEF)显著改善。与地拉罗司组相比,去铁酮组的全心 T2*改善显著降低。与去铁酮组相比,地拉罗司和去铁胺组的 LVEF 改善显著升高,而地拉罗司组的 RVEF 改善显著高于去铁酮组。在有基线肝铁的患者中,去铁酮与其他组相比,肝铁的变化相当。去铁酮单药治疗在改善心肌铁沉积和双心室功能方面不如地拉罗司有效,在改善 LVEF 方面不如去铁胺有效。

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