Approximately half the patients with autosomal dominant polycystic kidney disease (ADPKD), a condition due to deficiency in polycystin 1 or 2, have end-stage kidney disease by 60 years of age.^1,2 Once progression begins, the mean decline in the estimated glomerular filtration rate (GFR) is approximately 4 to 6 ml per minute per 1.73 m² of body-surface area per year. Therapy to prevent deterioration of GFR in patients with ADPKD has been elusive, despite mechanistic studies targeting pathogenesis. In addition, the burden of therapy in patients who have other symptoms from their ADPKD — hypertension, abdominal fullness and pain from cysts, . . .

中文翻译：

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托伐普坦和常染色体显性多囊肾

患有常染色体显性遗传性多囊肾病（ADPKD）的患者约有一半，这是由于多囊藻蛋白1或2缺乏引起的，到60岁时患有终末期肾脏病。^1,2一旦开始进展，估计的肾小球滤过率（GFR）的平均下降为每年每1.73 m ²体表面积每分钟约4至6 ml 。尽管有针对发病机理的机制研究，但防止ADPKD患者GFR恶化的疗法仍难以捉摸。此外，患有ADPKD症状的其他患者的治疗负担-高血压，腹胀和囊肿疼痛。。。