Pharmacological management of polycystic kidney disease
Pharmacological management of polycystic kidney disease
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DOI：
10.1517/14656566.2014.903923
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发表时间：
2014-05
影响因子：
3.2
通讯作者：
R. Wüthrich;C. Mei
中科院分区：
文献类型：
--
作者：
R. Wüthrich;C. Mei
简介：常染色体显性遗传性多囊肾病（ADPKD）代表了一种治疗挑战，因为尽管进行了数十年的密集基础和临床研究，但仍然没有有效的治疗方法来延缓肾脏和肝脏中囊肿的生长。涵盖的领域：近年来进行了几项临床试验，以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。已在随机临床试验中测试的药物类别包括雷帕霉素（mTOR）抑制剂的哺乳动物靶点西罗莫司和依维莫司，生长抑素类似物（奥曲肽，兰瑞肽，帕瑞肽），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。其他药物正在测试中，其中包括SRC-ABL酪氨酸激酶抑制剂博舒替尼和传统中药雷公藤内酯醇。延缓囊肿生长的其他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。专家意见：鉴于积累的知识，目前还不确定药物是否会在不久的将来变得可用，以显着改变无情进展的多囊肾疾病的进程。
Introduction: 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. Areas covered: 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. Expert opinion: 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.