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长期使用依库珠单抗治疗有助于非典型溶血性尿毒症综合征所致终末期肾病的恢复。

Long-term Eculizumab Treatment Contributes to Recovery from End-stage Renal Disease Caused by Atypical Hemolytic Uremic Syndrome.

作者信息

Yamada Yuji, Abe Ryohei, Okano Yutaka, Miyakawa Yoshitaka

机构信息

Department of Medicine, Kawasaki Municipal Kawasaki Hospital, Japan.

Department of Medicine, Mount Sinai Beth Israel, USA.

出版信息

Intern Med. 2017;56(9):1085-1088. doi: 10.2169/internalmedicine.56.7862. Epub 2017 May 1.

DOI:10.2169/internalmedicine.56.7862
PMID:28458317
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5478572/
Abstract

We experienced a favorable outcome in an adult case of atypical hemolytic uremic syndrome (aHUS) after long-term eculizumab treatment. A 38-year-old Japanese man with a history of central retinal vein occlusion was admitted to our hospital with progressive dyspnea. He was found to have non-immune hemolytic anemia, thrombocytopenia, and acute renal failure two weeks after an episode of the common cold. Plasma exchange was ineffective; therefore, we initiated eculizumab after we excluded other thrombotic microangiopathies. Although long-term peritoneal dialysis was required, we successfully discontinued dialysis 18 months after the onset of aHUS with eculizumab.

摘要

在一例成年非典型溶血尿毒综合征(aHUS)患者接受长期依库珠单抗治疗后,我们获得了良好的治疗效果。一名38岁有视网膜中央静脉阻塞病史的日本男性因进行性呼吸困难入院。他在普通感冒发作两周后被发现患有非免疫性溶血性贫血、血小板减少症和急性肾衰竭。血浆置换无效;因此,在排除其他血栓性微血管病后,我们开始使用依库珠单抗治疗。尽管需要长期进行腹膜透析,但在aHUS发病18个月后,我们成功停用了依库珠单抗。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b046/5478572/bed0d15dc6d1/1349-7235-56-1085-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b046/5478572/bed0d15dc6d1/1349-7235-56-1085-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b046/5478572/bed0d15dc6d1/1349-7235-56-1085-g001.jpg

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本文引用的文献

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Clinical guides for atypical hemolytic uremic syndrome in Japan.日本非典型溶血性尿毒症综合征临床指南
Pediatr Int. 2016 Jul;58(7):549-55. doi: 10.1111/ped.13044.
2
Eculizumab is a safe and effective treatment in pediatric patients with atypical hemolytic uremic syndrome.依库珠单抗对非典型溶血性尿毒症综合征患儿是一种安全有效的治疗方法。
Kidney Int. 2016 Mar;89(3):701-11. doi: 10.1016/j.kint.2015.11.026. Epub 2016 Jan 28.
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A novel quantitative hemolytic assay coupled with restriction fragment length polymorphisms analysis enabled early diagnosis of atypical hemolytic uremic syndrome and identified unique predisposing mutations in Japan.
一种新型定量溶血测定法与限制性片段长度多态性分析相结合,能够早期诊断非典型溶血尿毒症综合征,并在日本发现了独特的易感突变。
PLoS One. 2015 May 7;10(5):e0124655. doi: 10.1371/journal.pone.0124655. eCollection 2015.
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Role of the skin biopsy in the diagnosis of atypical hemolytic uremic syndrome.皮肤活检在非典型溶血尿毒综合征诊断中的作用
Am J Dermatopathol. 2015 May;37(5):349-56; quiz 357-9. doi: 10.1097/DAD.0000000000000234.
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Modified Ham test for atypical hemolytic uremic syndrome.非典型溶血性尿毒症综合征的改良哈姆试验
Blood. 2015 Jun 4;125(23):3637-46. doi: 10.1182/blood-2015-02-629683. Epub 2015 Apr 10.
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Syndromes of thrombotic microangiopathy.血栓性微血管病的综合征。
N Engl J Med. 2014 Aug 14;371(7):654-66. doi: 10.1056/NEJMra1312353.
7
Dynamics of complement activation in aHUS and how to monitor eculizumab therapy.补体激活在 aHUS 中的动力学及其如何监测依库珠单抗治疗。
Blood. 2014 Sep 11;124(11):1715-26. doi: 10.1182/blood-2014-02-558296. Epub 2014 Jul 18.
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Biomarkers of terminal complement activation confirm the diagnosis of aHUS and differentiate aHUS from TTP.末端补体激活的生物标志物可确诊 aHUS,并将其与 TTP 相区分。
Blood. 2014 Jun 12;123(24):3733-8. doi: 10.1182/blood-2013-12-547067. Epub 2014 Apr 2.
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