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对标准治疗方案反应欠佳的非典型溶血性尿毒症综合征患者依库珠单抗剂量模式的调整

Adjustment of Eculizumab Dosage Pattern in Patients with Atypical Hemolytic Uremic Syndrome with Suboptimal Response to Standard Treatment Pattern.

作者信息

García Monteavaro Camino, Peralta Roselló Carmen, Quiroga Borja, Baltar Martín José María, Castillo Eraso Lorena, de Álvaro Moreno Fernando, Martínez Vea Alberto, Visus-Fernández de Manzanos María Teresa

机构信息

Nephrology Service, Hospital San Agustín, Camino de Heros 6, 33401 Avilés, Spain.

Nephrology Service, Hospital Universitari Joan XXIII, C/Dr. Mallafrè Guasch 4, 43005 Tarragona, Spain.

出版信息

Case Rep Nephrol. 2016;2016:7471082. doi: 10.1155/2016/7471082. Epub 2016 Nov 29.

DOI:10.1155/2016/7471082
PMID:28025630
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5153492/
Abstract

In patients with atypical hemolytic uremic syndrome (aHUS), complement blocking by eculizumab rapidly halts the process of thrombotic microangiopathy and it is associated with clear long-term hematologic and renal improvements. Eculizumab treatment consists of a 4-week initial phase with weekly IV administration of 900 mg doses, followed by a maintenance phase with a 1,200 mg dose in the fifth week and every 14 ± 2 days thereafter. We present three patients with aHUS and suboptimal response to eculizumab treatment at the usual administration dosage who showed hematologic and renal improvements after an adjustment in the eculizumab treatment protocol.

摘要

在非典型溶血性尿毒症综合征(aHUS)患者中,依库珠单抗阻断补体可迅速终止血栓性微血管病进程,并与明显的长期血液学和肾脏改善相关。依库珠单抗治疗包括一个为期4周的初始阶段,每周静脉注射900毫克剂量,随后是维持阶段,在第五周注射1200毫克剂量,此后每14±2天注射一次。我们报告了3例aHUS患者,他们在常规给药剂量下对依库珠单抗治疗反应欠佳,但在调整依库珠单抗治疗方案后出现了血液学和肾脏改善。

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An update for atypical haemolytic uraemic syndrome: diagnosis and treatment. A consensus document.非典型溶血性尿毒症综合征的最新进展:诊断与治疗。一份共识文件。
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Assessing complement blockade in patients with paroxysmal nocturnal hemoglobinuria receiving eculizumab.评估接受依库珠单抗治疗的阵发性睡眠性血红蛋白尿症患者的补体阻断情况。
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[Diagnostic and therapeutic guidelines of thrombotic microangiopathies of the Spanish Apheresis Group].[西班牙血液成分单采小组血栓性微血管病的诊断与治疗指南]
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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。
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Genetic variants in C5 and poor response to eculizumab.C5 中的遗传变异与依库珠单抗反应不佳有关。
N Engl J Med. 2014 Feb 13;370(7):632-9. doi: 10.1056/NEJMoa1311084.