Wüthrich Rudolf P, Mei Changlin
University Hospital, Division of Nephrology , Rämistrasse 100, 8091 Zürich , Switzerland +41 44 255 33 84 ; +41 44 255 45 93 ;
Expert Opin Pharmacother. 2014 Jun;15(8):1085-95. doi: 10.1517/14656566.2014.903923. Epub 2014 Mar 28.
Autosomal-dominant polycystic kidney disease (ADPKD) represents a therapeutic challenge as effective treatment to retard the growth of cysts in the kidneys and the liver has not been available despite decades of intense basic and clinical research.
Several clinical trials have been performed in recent years to study the effect of diverse drugs on the growth of renal and hepatic cysts, and on functional deterioration of the glomerular filtration rate. The drug classes that have been tested in randomized clinical trials include the mammalian target of rapamycin (mTOR) inhibitors, sirolimus and everolimus, the somatostatin analogues (octreotide, lanreotide, pasireotide), and most recently, the vasopressin V2 receptor antagonist, tolvaptan. The results with the mTOR inhibitors were disappointing, but more encouraging with the somatostatin analogues and with tolvaptan. Additional drugs are being tested, which include among others, the SRC-ABL tyrosine kinase inhibitor, bosutinib, and the traditional Chinese herbal medication, triptolide. Additional therapeutic strategies to retard cyst growth aim at blood pressure control via inhibition of the renin-angiotensin system and the sympathetic nervous system.
Given the accumulated knowledge, it is currently uncertain whether drugs will become available in the near future to significantly change the course of the relentlessly progressing polycystic kidney disease.
常染色体显性遗传性多囊肾病（ADPKD）是一项治疗挑战，尽管经过数十年深入的基础和临床研究，但仍未找到有效延缓肾囊肿和肝囊肿生长的治疗方法。
近年来开展了多项临床试验，研究不同药物对肾囊肿和肝囊肿生长以及肾小球滤过率功能恶化的影响。在随机临床试验中测试过的药物类别包括雷帕霉素靶蛋白（mTOR）抑制剂西罗莫司和依维莫司、生长抑素类似物（奥曲肽、兰瑞肽、帕西瑞肽），以及最近的加压素V2受体拮抗剂托伐普坦。mTOR抑制剂的试验结果令人失望，但生长抑素类似物和托伐普坦的结果更令人鼓舞。正在测试其他药物，其中包括SRC-ABL酪氨酸激酶抑制剂博舒替尼和传统中草药雷公藤内酯醇。其他延缓囊肿生长的治疗策略旨在通过抑制肾素-血管紧张素系统和交感神经系统来控制血压。
鉴于已积累的知识，目前尚不确定近期是否会有药物问世，从而显著改变多囊肾病不断进展的病程。
