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地拉罗司(Exjade)可显著改善重型β-地中海贫血患者的心脏 T2*。

Deferasirox (Exjade) significantly improves cardiac T2* in heavily iron-overloaded patients with beta-thalassemia major.

机构信息

Sultan Qaboos University Hospital, Muscat, Oman.

出版信息

Ann Hematol. 2010 Apr;89(4):405-9. doi: 10.1007/s00277-009-0838-z. Epub 2009 Oct 2.

DOI:10.1007/s00277-009-0838-z
PMID:19798501
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC2824843/
Abstract

Noninvasive measurement of tissue iron levels can be assessed using T2* magnetic resonance imaging (MRI) to identify and monitor patients with iron overload. This study monitored cardiac siderosis using T2* MRI in a cohort of 19 heavily iron-overloaded patients with beta-thalassemia major receiving iron chelation therapy with deferasirox over an 18-month period. Overall, deferasirox therapy significantly improved mean + or - standard deviation cardiac T2* from a baseline of 17.2 + or - 10.8 to 21.5 + or - 12.8 ms (+25.0%; P = 0.02). A concomitant reduction in median serum ferritin from a baseline of 5,497 to 4,235 ng/mL (-23.0%; P = 0.001), and mean liver iron concentration from 24.2 + or - 9.0 to 17.6 + or - 12.9 mg Fe/g dry weight (-27.1%; P = 0.01) was also seen. Improvements were seen in patients with various degrees of cardiac siderosis, including those patients with a baseline cardiac T2* of <10 ms, indicative of high cardiac iron burden. These findings therefore support previous observations that deferasirox is effective in the removal of myocardial iron with concomitant reduction in total body iron.

摘要

组织铁水平的无创测量可使用 T2磁共振成像(MRI)进行评估,以识别和监测铁过载患者。本研究在 19 名接受地拉罗司铁螯合治疗的重型β-地中海贫血患者队列中使用 T2MRI 监测心脏含铁血黄素沉着症,治疗时间为 18 个月。总体而言,地拉罗司治疗显著改善了心脏 T2的平均值(+/-标准差),从基线的 17.2+/-10.8 增加到 21.5+/-12.8 ms(增加 25.0%;P=0.02)。同时,血清铁蛋白中位数从基线的 5497 降至 4235 ng/mL(减少 23.0%;P=0.001),肝脏铁浓度的平均值从 24.2+/-9.0 降至 17.6+/-12.9 mg Fe/g 干重(减少 27.1%;P=0.01)。心脏 siderosis 程度不同的患者均观察到改善,包括基线心脏 T2<10 ms 的患者,这表明心脏铁负荷较高。这些发现支持了先前的观察结果,即地拉罗司可有效去除心肌铁,同时减少全身铁含量。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6f51/2824843/2cabc359c34a/277_2009_838_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6f51/2824843/2cabc359c34a/277_2009_838_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6f51/2824843/2cabc359c34a/277_2009_838_Fig1_HTML.jpg

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Efficacy and safety of deferasirox, an oral iron chelator, in heavily iron-overloaded patients with beta-thalassaemia: the ESCALATOR study.口服铁螯合剂地拉罗司对重度铁过载β地中海贫血患者的疗效和安全性:ESCALATOR研究
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Health-related quality of life (HRQoL) in beta-thalassemia major (β-TM) patients assessed by 36-item short form health survey (SF-36): a meta-analysis.采用 36 项简明健康调查问卷(SF-36)评估重型β-地中海贫血患者的健康相关生活质量(HRQoL):一项荟萃分析。
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Transplantation in patients with iron overload: is there a place for magnetic resonance imaging? : Transplantation in iron overload.铁过载患者的移植:磁共振成像有一席之地吗?:铁过载中的移植。
Heart Fail Rev. 2018 Mar;23(2):173-180. doi: 10.1007/s10741-018-9670-7.
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Effect of iron overload on impaired fertility in male patients with transfusion-dependent beta-thalassemia.铁过载对输血依赖型β-地中海贫血男性患者生育能力受损的影响。
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