常染色体显性遗传多囊肾病药物治疗研究进展  

Research progress of drug therapy for ADPKD

作　　者：牛青松 徐雨辰[1] 马东玥 柏争鸣 郝宗耀[1] 梁朝朝(审校)[1] NIU Qingsong;XU Yuchen;MA Dongyue;BAI Zhengming;HAO Zongyao;LIANG Chaozhao(Department of Urology,First Affiliated Hospital of Anhui Medical University,Institute of Urology,Anhui Medical University,Anhui Province Key Laboratory of Genitourinary Diseases,Anhui Province PKD Center,Hefei,230022,China)

机构地区：[1]安徽医科大学第一附属医院泌尿外科,安徽医科大学泌尿外科研究所,泌尿生殖系统疾病安徽省重点实验室,安徽省多囊肾诊疗与转化医学中心,合肥230022

出　　处：《临床泌尿外科杂志》2020年第10期842-845,共4页Journal of Clinical Urology

基　　金：安徽省国际科技合作计划项目(No:1704e1002230)。

摘　　要：常染色体显性遗传多囊肾病(ADPKD)是一种单基因遗传性肾脏疾病。ADPKD的病理特征是肾脏多发囊肿的进行性生成和增长。增大的囊肿破坏肾实质,损害肾功能,最终导致终末期肾病(ESRD)。目前临床上仍缺乏阻止ADPKD疾病进展的有效药物,对症治疗是ADPKD的主要治疗方式,包括降压、止痛、透析等。近年来,一些有望治疗ADPKD的潜在药物不断出现。本文主要综述ADPKD的临床和临床前治疗药物试验研究进展。Autosomal dominant polycystic kidney disease(ADPKD)is a monogenic inherited kidney disease.ADPKD is characterized by progressive cyst formation and growth in bilateral kidney.The renal lesion will result in end-stage renal disease(ESRD).Currently,there is still a lack of effective drugs to prevent the progression of ADPKD.Symptomatic treatment,including anti-hypertension,pain relief and renal replacement therapy are the main methods used in clinic.In recent years,potential drugs for ADPKD are emerging,and this article centered on the progress in the clinical and preclinical researches of ADPKD.

关 键 词：常染色体显性遗传多囊肾病 治疗药物 临床试验 临床前试验 

分 类 号：R691[医药卫生—泌尿科学]