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联合治疗——地拉罗司与去铁胺——用于资源有限的新兴国家的重型地中海贫血患者。

Combination therapy - deferasirox and deferoxamine - in thalassemia major patients in emerging countries with limited resources.

作者信息

Arandi N, Haghpanah S, Safaei S, Zahedi Z, Ashrafi A, Eatemadfar P, Zarei T, Radwan A H, Taher A T, Karimi M

机构信息

Hematology Research Center, Shiraz University of Medical Sciences, Shiraz, Iran.

出版信息

Transfus Med. 2015 Feb;25(1):8-12. doi: 10.1111/tme.12188. Epub 2015 Mar 19.

Abstract

BACKGROUND

The problem of iron-overload observed in thalassemia patients can be overcome using chelating agents such as deferiprone (Ferroprox(®) ), deferasirox (Exjade(®) ) and deferoxamine (Desferal(®) ). Although these drugs can be used as monotherapy, combined therapy, especially deferiprone with deferoxamine, has led to promising outcomes in various studies.

METHODS AND MATERIALS

In this quasi-experimental study, serum ferritin levels were evaluated in 32 β-thalassemia major patients with severe iron overload before and after receiving combined deferasirox (30-40 mg kg(-1)  day(-1) ) and deferoxamine (40-50 mg kg(-1)  day(-1) ) 2 days a week. This study was conducted from September 2012 to September 2013 in Southern Iran.

RESULTS

The mean of serum ferritin levels significantly reduced from 4031 ± 1955 to 2416 ± 1653 ng mL(-1) after 12 months of therapy (P < 0·001). Echocardiograph findings showed significant improvement 1year after end of the study (P < 0·001). No drug toxicity was observed by monitoring serum creatinine, liver enzymes and blood urea nitrogen (BUN) during the study period. We observed no correlation between mean serum ferritin change and age (P = 0·87). In addition, the mean serum ferritin change did not differ between male and female thalassemia patients (P = 0·454). No difference in mean serum ferritin change was observed between patients who had undergone splenectomy compared to those who had not done so (P = 0·307).

CONCLUSION

The study suggests that combination chelating therapy with deferasirox and deferoxamine can effectively reduce iron burden in β-thalassemia major patients with heavy iron overload without any significant complications.

摘要

背景

地中海贫血患者中观察到的铁过载问题可通过使用去铁酮(Ferroprox®)、地拉罗司(Exjade®)和去铁胺(Desferal®)等螯合剂来克服。尽管这些药物可作为单一疗法使用,但联合疗法,尤其是去铁酮与去铁胺联合使用,在各项研究中已取得了有前景的结果。

方法和材料

在这项准实验研究中,对32例重度铁过载的β-地中海贫血重型患者在接受联合地拉罗司(30 - 40 mg kg⁻¹ 天⁻¹)和去铁胺(40 - 50 mg kg⁻¹ 天⁻¹)每周2天治疗前后的血清铁蛋白水平进行了评估。本研究于2012年9月至2013年9月在伊朗南部进行。

结果

治疗12个月后,血清铁蛋白水平均值从4031±1955显著降至2416±1653 ng mL⁻¹(P<0.001)。超声心动图检查结果显示在研究结束1年后有显著改善(P<0.001)。在研究期间,通过监测血清肌酐、肝酶和血尿素氮(BUN)未观察到药物毒性。我们观察到血清铁蛋白平均变化与年龄之间无相关性(P = 0.87)。此外,男性和女性地中海贫血患者的血清铁蛋白平均变化无差异(P = 0.454)。与未进行脾切除术的患者相比,进行脾切除术的患者血清铁蛋白平均变化无差异(P = 0.307)。

结论

该研究表明,地拉罗司和去铁胺联合螯合疗法可有效降低重度铁过载的β-地中海贫血重型患者的铁负荷,且无任何显著并发症。

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