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首页> 外文期刊>Pediatric nephrology: journal of the International Pediatric Nephrology Association >Eculizumab therapy for atypical haemolytic uraemic syndrome due to a gain-of-function mutation of complement factor B
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Eculizumab therapy for atypical haemolytic uraemic syndrome due to a gain-of-function mutation of complement factor B

机译：Eculizumab治疗因补体因子B功能获得性突变而引​​起的非典型溶血性尿毒症综合征

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摘要

Background: Atypical haemolytic uraemic syndrome (aHUS) is caused by dysregulated complement activation. A humanised anti-C5 monoclonal antibody has recently become available for treatment of this condition Case-Diagnosis/ Treatment: We present the first description of an infant with an activating mutation of complement factor B successfully treated with eculizumab. On standard doses she had evidence of ongoing C5 cleavage despite a good clinical response. Conclusions: Eculizumab is effective therapy for aHUS associated with factor B mutations, but recommended doses may not be adequate for all patients.
机译：背景：非典型溶血性尿毒症综合征（aHUS）是由补体激活失调引起的。人源化抗C5单克隆抗体最近已可用于治疗这种情况。病例诊断/治疗：我们首次描述了用依库丽单抗成功治疗的具有补体因子B激活突变的婴儿。尽管有良好的临床反应，但在标准剂量下，她有持续进行C5裂解的证据。结论：依库丽单抗是治疗与因子B突变相关的aHUS的有效疗法，但推荐剂量可能并不适合所有患者。

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