Galliani Marco, Chicca Silvana, Vitaliano Elio, Moscaritolo Eleonora, Calvaruso Luca, Iorio Francesco, Paone Antonio
UOC Nefrologia, Dialisi e Litotrissia, Ospedale Sandro Pertini Roma.
UOC Nefrologia, Fondazione Policlinico Universitario A. Gemelli IRCCS, Università Cattolica del Sacro Cuore, Roma.
G Ital Nefrol. 2019 Dec 9;36(6):2019-vol6.
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most frequent monogenic hereditary disease as well as the most studied inherited kidney disease. Two drugs have recently been authorized that can slow down the progression of the disease: Tolvaptan (vasopressin receptor antagonist) and Octreotide-LAR (long-acting somatostatin analogue); they both are able to reduce the activity of cyclic adenosine monophosphate (cAMP) and therefore have anti-proliferative and anti-secretory effects. This review analyzes the main trials published to date demonstrating the effects on disease progression in patients with ADPKD and illustrates the indications for identifying subjects eligible for therapy.
常染色体显性多囊肾病(ADPKD)是最常见的单基因遗传性疾病,也是研究最多的遗传性肾脏疾病。最近有两种药物已获批准,可减缓该疾病的进展:托伐普坦(血管加压素受体拮抗剂)和长效奥曲肽(长效生长抑素类似物);它们都能够降低环磷酸腺苷(cAMP)的活性,因此具有抗增殖和抗分泌作用。本综述分析了迄今为止发表的主要试验,这些试验证明了对ADPKD患者疾病进展的影响,并阐述了识别适合治疗的受试者的指征。