Atypical hemolytic uremic syndrome (aHUS), is mainly present in children, who have high risks of end-stage kidney disease (ESKD), post-transplant recurrence and death. aHUS is linked to defective regulation of the complement alternative pathway (AP), with a prominent cause being mutation/inhibition of the negative regulator complement factor H (CFH). CFH function can be restored via infusion of fresh frozen plasma (FFP), a treatment that was effective for several years in a patient heterozygous for a cfh mutation, before the patient progressed to ESKD. While on dialysis, FFP was replaced with eculizumab, which blocks C5 cleavage and thus halts progression of the terminal complement pathway. Patient plasma samples collected during FFP and eculizumab treatment phases were assessed for AP activity (via erythrocyte lysis assays) and for overall complement activity (via ELISA-based screen). Assay results indicated that FFP partially restored AP regulation, an observation supported by in vitro modeling of FFP treatment using purified CFH, while eculizumab completely blocked complement activity. The same approach was used to model in vitro a potential aHUS treatment approach based on blocking the AP effector properdin (complement factor P; CFP) with an anti-properdin antibody. These results provide insights into the efficacy of aHUS treatment and highlight the usefulness of in vitro assays in monitoring and predicting therapeutic responses and testing new treatment possibilities.

译文

:非典型溶血性尿毒症综合征(aHUS)主要存在于儿童中,这些儿童罹患终末期肾脏疾病(ESKD),移植后复发和死亡的风险很高。 aHUS与补体替代途径(AP)的调控缺陷有关,其主要原因是负调控因子补体因子H(CFH)的突变/抑制。可以通过输注新鲜冷冻血浆(FFP)恢复CFH功能,这种治疗对杂散的cfh突变的患者有效了数年,在患者发展为ESKD之前。透析时,FFP被依库丽单抗替代,依古丽单抗阻止C5裂解,因此终止了末端补体途径的进程。在FFP和依库丽单抗治疗阶段收集的患者血浆样品的AP活性(通过红细胞裂解测定)和总体补体活性(通过基于ELISA的筛查)被评估。分析结果表明,FFP部分恢复了AP的调节作用,这一观察结果得到了使用纯化CFH进行FFP治疗的体外模型支持,而依库丽单抗则完全阻断了补体活性。基于使用抗备解素抗体阻断AP效应素备解素(补体因子P; CFP)的方法,使用相同的方法在体外对潜在的aHUS治疗方法进行建模。这些结果提供了对aHUS治疗功效的见解,并突出了体外测定法在监测和预测治疗反应以及测试新治疗可能性方面的有用性。

+1
+2
100研值 100研值 ¥99课程
检索文献一次
下载文献一次

去下载>

成功解锁2个技能,为你点赞

《SCI写作十大必备语法》
解决你的SCI语法难题!

技能熟练度+1

视频课《玩转文献检索》
让你成为检索达人!

恭喜完成新手挑战

手机微信扫一扫,添加好友领取

免费领《Endnote文献管理工具+教程》

微信扫码, 免费领取

手机登录

获取验证码
登录