抗体介导的aHUS和TTP的免疫抑制疗法
Kata Kelen1, Orsolya Horváth1, Éva Kis2
1Bókay Street Unit, Department of Pediatrics, Semmelweis University, 1083 Budapest, Hungary.
International journal of molecular sciences
|September 28, 2023
概括
在儿童中,抗体介导的血栓性微血管病变 (TMA),包括非典型的血溶性尿素性综合征 (aHUS) 和血栓性血栓细胞性紫外线 (TTP),可以通过血交换和早期免疫抑制实现持续缓解. 长期结果显示功能得到保护,但潜在的晚期并发症.
科学领域:
- 儿科脏病学 儿科脏病学
- 血液学 血液学 血液学
- 免疫学 免疫学 免疫学
背景情况:
- 血栓式微血管病变 (TMA) 的分类现在包括针对性治疗的疾病机制.
- 抗体介导的TMA,包括非典型的血溶性尿素综合征 (aHUS) 和血栓性血栓塞性紫外线 (TTP),在儿科患者中存在独特的挑战.
研究的目的:
- 报告七名患有抗体介导的aHUS和TTP的儿科患者的经验.
- 评估这些疾病治疗方案的疗效和长期结果.
主要方法:
- 五名患者患有抗补因子H抗体 (CFH-ab) 的aHUS;两名患有抗ADAMTS13抗体的TTP.
- 诊断方法包括CFH-ab的ELISA和ADAMTS13活动的评估.
- 治疗包括血交换,类固醇脉冲和用环胺和修复剂抑制免疫力.
主要成果:
- 四名患有CFH-ab的aHUS患者和两名TTP患者实现了持续的缓解.
- 在没有维持治疗的中位数随访11.7年后,没有观察到疾病复发.
- 晚期并发症包括一些患者的高血压和蛋白尿; 一名患者患上末期病.
结论:
- 血交换和早期免疫抑制治疗在抗体介导的TMA中有效地实现持续缓解和保持功能.
- 需要进行进一步的研究,以优化对抗FH抗体相关的HUS.US.治疗策略.
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