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利用生物磁肝脏磁测量法监测铁螯合治疗的长期疗效。

Monitoring long-term efficacy of iron chelation treatment with biomagnetic liver susceptometry.

作者信息

Fischer Roland, Piga Antonio, Harmatz Paul, Nielsen Peter

机构信息

University Medical Center Hamburg-Eppendorf, Department of Molecular Cell Biology, Hamburg, Germany.

出版信息

Ann N Y Acad Sci. 2005;1054:350-7. doi: 10.1196/annals.1345.043.

Abstract

In patients with thalassemia, the assessment of liver iron concentration (LIC) can be used to initiate chelation treatment with desferrioxamine (DFO), deferiprone (DFP), or novel chelators (deferasirox); to adjust chelation dose according to the actual blood transfusion rate; and to monitor chelation efficacy. The results from measurements by SQUID biomagnetic liver susceptometry in the LIC range 17-11,500 microg/g of liver in about 1000 patients were used to derive nonstandard parameters, which may be useful in the treatment monitoring of patients with thalassemia. From these measurements, including liver volumes, the documented chelation dose rates, and the blood transfusion rates, the chelator index (equivalent Therapeutical Index), the total body iron elimination rate, and the molar efficacy were calculated. Chelator indices (CIs) ranged from 0.1 to 11.7 mmol/d/g of Fe for DFO, with a threshold of CI greater than 1.2 mmol/d/g of Fe indicating DFO toxicity. For DFP, CI ranged from 0.1 to 23.2 mmol/d/g of Fe. In long-term studies (2 and 4 years), mean molar efficacies of DFO and DFP were found to be quite stable with 17.6 +/- 4.8% and 4.9 +/- 1.4%, respectively. Currently, specific chelation dose is based upon body weight. Because liver iron measurements by biosusceptometry are now regularly available in Europe and America, as well as quantitative MRI worldwide, these methods may be used to adjust chelation treatment regimens to body iron stores.

摘要

对于地中海贫血患者,评估肝脏铁浓度(LIC)可用于启动去铁胺(DFO)、地拉罗司(DFP)或新型螯合剂(地拉罗司)的螯合治疗;根据实际输血率调整螯合剂量;并监测螯合疗效。通过超导量子干涉装置生物磁肝脏磁测量法对约1000例患者进行测量,测量范围为肝脏LIC在17 - 11500μg/g之间,由此得出的非标准参数可能有助于地中海贫血患者的治疗监测。从这些测量结果,包括肝脏体积、记录的螯合剂量率和输血率,计算出螯合剂指数(等效治疗指数)、全身铁清除率和摩尔疗效。DFO的螯合剂指数(CIs)范围为0.1至11.7 mmol/d/g Fe,当CI阈值大于1.2 mmol/d/g Fe时表明存在DFO毒性。对于DFP,CI范围为0.1至23.2 mmol/d/g Fe。在长期研究(2年和4年)中,发现DFO和DFP的平均摩尔疗效相当稳定,分别为17.6±4.8%和4.9±1.4%。目前,具体的螯合剂量是基于体重的。由于目前在欧美地区通过生物磁测量法进行肝脏铁测量已常规可用,以及在全球范围内定量MRI也已可用,这些方法可用于根据体内铁储存量调整螯合治疗方案。

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