常染色體顯性多囊腎病(ADPKD)是一種遺傳性腎囊腫性疾病,臨床中缺乏特異性治療藥物,治療重點(diǎn)在于緩解癥狀,若發(fā)展為腎功能衰竭則需采取透析、腎移植等替代治療。為進(jìn)一步開展治療ADPKD的臨床試驗(yàn)及應(yīng)用提供參考,查閱最新國內(nèi)外研究報道,分析總結(jié)了目前治療ADPKD的藥物主要有血管加壓素V2受體(AVPV2R)拮抗劑托伐普坦、哺乳動物雷帕霉素靶蛋白(mTOR)抑制劑西羅莫司和依維莫司、生長抑素類似物(SST)奧曲肽、他汀類藥物洛伐他汀和普伐他汀進(jìn)入臨床試驗(yàn)階段,其中AVPV2R拮抗劑托伐普坦已在日本被批準(zhǔn)為世界首個用于延緩ADPKD進(jìn)展的治療藥物,但由于其不良反應(yīng)尚未被FDA正式批準(zhǔn),大規(guī)模臨床試驗(yàn)和隨后的風(fēng)險–效益分析有待進(jìn)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.