在焦点细分细胞凝结症的任何时间完全蛋白尿缓解的影响:Sparsentan DUET试验
Howard Trachtman1, Ulysses Diva2, Edward Murphy2
1Division of Nephrology, Department of Pediatrics, University of Michigan, Ann Arbor, Michigan, USA.
在焦点细分细胞凝聚硬化 (FSGS) 中,用斯帕森坦实现蛋白尿完全缓解,即使是暂时的,也与更好的长期功能有关. 这表明sparsentan的存在.
科学领域:
- 腎臟病學 (nephrology) 是一種醫學專業.
- 淋巴细胞疾病 淋巴细胞疾病
- 临床试验 临床试验
背景情况:
- 焦点细分性淋巴结核硬化 (FSGS) 是一种罕见的病,治疗选择有限.
- 蛋白尿是FSGS治疗疗效的关键标志物.
- 蛋白尿完全缓解 (CR) 是新疗法的潜在替代终点.
研究的目的:
- 评估FSGS患者中至少实现1次蛋白尿的CR和长期结局之间的关系,这些患者接受了sparsentan治疗.
- 在FSGS中评估sparsentan的安全性和持续的抗蛋白尿作用.
主要方法:
- 从DUET试验及其开放标签扩展 (OLE) 中对接受sparsentan治疗的患者进行后期分析.
- 患者每12周评估一次血压,,蛋白尿和功能.
- CR被定义为尿蛋白/肌素比率≤0.3g/g.
主要成果:
- 在108名患者中,43%的患者在47.0个月的中位随访期间至少一次达到CR.
- 达到CR与估计球过率 (eGFR) 降低的速度显著减缓有关.
- 斯帕森坦在同时接受免疫抑制治疗时显示出添加剂抗蛋白尿效应,没有新的安全问题.
结论:
- 斯帕森坦可以安全地长期使用,表现出持续的抗蛋白尿作用.
- 实现CR,即使不持续,也预测了对sparsentan的良好反应和FSGS中改善的结局.
- 蛋白尿症缓解是评估FSGS治疗有效性的重要指标.
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