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晚期发病的血小血管病在脏移植中; 尽管治疗了eculizumab,但结果不佳
Emily K Glover1,2, Emma K Montgomery1, Edwin K S Wong1,2
1National Renal Complement Therapeutics Centre, Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom.
概括
在非典型的血溶性尿素综合征 (aHUS) 患者中,治疗移植后的血栓性微血管病变 (TMA) 的eculizumab治疗,在及时开始时显示出改善的结果. 移植后12个月以上的延迟治疗显著恶化了移植的存活率.
科学领域:
- 腎臟病學 (nephrology) 是一種醫學.
- 移植免疫学 移植免疫学
- 罕见疾病 罕见疾病
背景情况:
- 非典型的血溶性尿素综合征 (aHUS) 是末期病 (ESKD) 的罕见原因.
- 移植后HUS复发可能导致血栓性微血管病变 (TMA).
- 埃奎利祖马布是与HUS相关的TMA的潜在预防或治疗剂.
研究的目的:
- 为了评估eculizumab在移植后TMA患者的疗效,由于aHUS.
- 根据与移植相对的eculizumab启动时间来评估结果.
主要方法:
- 对26名患者进行了回顾性病例笔记审查,这些患者接受了eculizumab治疗,用于移植后的TMA.
- 查补充通路缺陷和抗H因子自身抗体.
- 在开始eculizumab治疗12个月后对死亡审查的移植存活的分析.
主要成果:
- 34.6%的接受者发现了补充通路缺陷.
- 移植后TMA呈现的中位时间为移植后8.4个月.
- 总体而言,12个月死亡审查的移植存活率为68%;然而,在移植后12个月以上的患者中,仅为42.9%.
结论:
- 埃奎利祖马布可以用于治疗aHUS患者的移植后TMA.
- 及时开始治疗 (移植后<12个月) 与更好的移植存活率有关.
- 移植后12个月以上的TMA患者的结局仍然很差,即使是使用eculizumab.
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