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《新英格兰医学杂志》2012年12月发表的一项多中心、双盲、安慰剂对照研究表明,托伐坦(Tolvaptan)用于治疗常染色体显性多囊肾病(autosomal dominant polycystic kidney disease,ADPKD),可以明显延缓肾脏总体积增大和肾功能减退。随着ADPKD的病情进展,患者常并发疼痛、高血压和肾功能衰竭。临床前试验表明,血管加压素V2受体拮抗剂可抑制肾囊肿的生长,延缓肾功能的下降。在这个三期、多中心、双盲、安慰剂对照的试验中,研究人员将1445例18~50岁的ADPKD(肾脏总体积≥750 ml,估算的肌酐清除率≥60 ml/min)患者随机分配,以2∶1的比例分别接受V2受体拮抗剂托伐坦和安慰剂的治疗。在病人可耐受的情况下,按每日两次的最大剂量给予托伐坦或安慰剂。主要终点是每年肾脏总体积的变化率。次要终点为临床进展(包括肾功能恶化、肾区疼痛、高血压、蛋白尿)的复合终点和肾功能减退率。经过了3年的研究,托伐坦组的肾脏总体积增长速度是每年2.8%(95%CI=2.5~3.1),安慰剂组肾脏总体积的增长速度是每年5.5%(95%CI=2.1~6.1;P<0.001)。
A multicenter, double-blind, placebo-controlled study published in the New England Journal of Medicine in December 2012 showed that Tolvaptan is used in the treatment of autosomal dominant polycystic kidney disease (ADPKD ), Can significantly delay the total volume of the kidneys and renal dysfunction. With the progression of ADPKD, patients often develop pain, hypertension, and renal failure. Preclinical studies have shown that vasopressin V2 receptor antagonists can inhibit the growth of renal cysts and delay the decline of renal function. In a three-phase, multicenter, double-blind, placebo-controlled trial, the researchers randomized 1445 ADPKD patients 18 to 50 years old (total kidney volume ≥750 ml, estimated creatinine clearance ≥60 ml / min) Allocated, in a 2: 1 ratio, received the V2 receptor antagonist tovavastatin and placebo. In patients tolerated, atorvastatin or placebo was administered at the maximum dose twice daily. The primary endpoint is the rate of change of total kidney volume per year. Secondary endpoints were composite endpoints of clinical progression (including worsening renal function, pain in the kidney area, hypertension, proteinuria) and rates of renal dysfunction. After 3 years of study, the total volume of kidney in the atorvastatin group grew at a rate of 2.8% per year (95% CI = 2.5 to 3.1) and 5.5% (95% CI = 2.1) per year for the placebo group ~ 6.1; P <0.001).