Burtey Stéphane
Hôpital de la conception, centre de néphrologie et transplantation rénale, 13005 Marseille, France.
Presse Med. 2011 Nov;40(11):1059-64. doi: 10.1016/j.lpm.2011.08.001. Epub 2011 Oct 2.
Autosomal dominant polycystic kidney disease is the most frequent renal genetic disease. Its main complication is renal failure. Despite a better understanding of the mechanisms leading to cyst development and growth, no specific treatment is available. Inhibition of mTOR pathway was a great hope, unfortunately, two clinical trials failed to show a clinical benefit. Numerous new drugs are in clinical trials or in the pipe-line. We could hope, in the 5 years to the emergence of an efficient treatment to slow the pace of renal failure in ADPKD.
常染色体显性多囊肾病是最常见的肾脏遗传性疾病。其主要并发症是肾衰竭。尽管对导致囊肿形成和生长的机制有了更深入的了解,但仍没有特效治疗方法。抑制mTOR通路曾带来很大希望,遗憾的是,两项临床试验未能显示出临床益处。许多新药正在进行临床试验或处于研发阶段。我们有望在未来5年内出现一种有效的治疗方法,以减缓常染色体显性多囊肾病患者肾衰竭的进展速度。