伊普塔科潘可降低蛋白尿和稳定功能在C3球粒发病
Carla M Nester1, Ute Eisenberger2, Alexandre Karras3
1Stead Family Children's Hospital-University of Iowa, Iowa City, Iowa, USA.
伊普塔科潘显著改善功能,并减少了C3型血小板病 (C3G) 患者的蛋白尿症. 这种口服补充抑制剂在治疗这种罕见的病方面表现有前途.
科学领域:
- 腎臟病學 (nephrology) 是一種醫學專業.
- 补充系统生物学 补充系统生物学
- 药理学 药理学是指药理学的学科.
背景情况:
- C3球囊病 (C3G) 是一种罕见的,进展性病,由替代补充路径过度激活引起.
- 它经常导致功能衰竭和移植复发.
- 伊普塔科潘是一种口服补充抑制剂,向B因子,选择性地抑制替代途径.
研究的目的:
- 评估伊普塔科潘在患有C3G的成年患者中的疗效和安全性.
- 评估伊普塔科潘对蛋白尿,功能和补体沉积的影响.
主要方法:
- 在26名成年患者的第二阶段扩展研究中,本土C3G (A队列) 或移植后复发的C3G (B队列).
- 患者接受了开放标签的iptacopan.
- 评估包括24小时尿蛋白与肌素比率 (UPCR),估计的淋巴细胞过率 (eGFR) 和12个月的血清C3水平.
主要成果:
- 在A队列中,iptacopan导致UPCR减少57%,并在12个月后改善了eGFR6.83毫升/分钟/1.73米2.
- 在两个队列中,血清C3水平显著增加.
- 在B队列中,虽然蛋白尿减少很小,但血清C3水平显著增加,EGFR保持稳定.
结论:
- 伊普塔科潘在C3G患者中显示出显著的临床益处,包括减少蛋白尿和改善功能.
- 这项研究支持进一步调查长期治疗C3G.伊普塔科潘的研究.
- 伊普塔科潘为这种极为罕见的病提供了潜在的治疗选择.
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