常染色体显性多囊肾病(ADPKD)是一种遗传性肾囊肿性疾病,临床中缺乏特异性治疗药物,治疗重点在于缓解症状,若发展为肾功能衰竭则需采取透析、肾移植等替代治疗。为进一步开展治疗ADPKD的临床试验及应用提供参考,查阅最新国内外研究报道,分析总结了目前治疗ADPKD的药物主要有血管加压素V2受体(AVPV2R)拮抗剂托伐普坦、哺乳动物雷帕霉素靶蛋白(mTOR)抑制剂西罗莫司和依维莫司、生长抑素类似物(SST)奥曲肽、他汀类药物洛伐他汀和普伐他汀进入临床试验阶段,其中AVPV2R拮抗剂托伐普坦已在日本被批准为世界首个用于延缓ADPKD进展的治疗药物,但由于其不良反应尚未被FDA正式批准,大规模临床试验和随后的风险–效益分析有待进一步深入研究。

Autosomal dominant polycystic kidney disease (ADPKD) is a hereditary renal cystic disease. For lack of specific clinical therapy, it focuses on relieving the symptoms. If ADPKD develops to end-stage renal failure, patients need to take replacement therapy, such as dialysis, kidney transplantation. Therefore, in order to provide a reference for carrying on further clinical trials and treatment for ADPKD, the latest domestic and foreign research reports are reviewed and summarized. So far, there are vasopressin V2 receptor (AVPV2R) antagonists tolvaptan, mammalian target of rapamycin inhibitors sirolimus and everolimus, somatostatin analogues octreotide, and statins lovastatin and pravastatin in clinical trials, in which AVPV2R antagonist tolvaptan has been approved for the first drug in the world to delay the progression of ADPKD in Japan. But because of the adverse reactions, tolvaptan has not yet been formally approved by FDA. Large-scale clinical trials and subsequent risk-benefit analysis need to be further studied.