Rysz Jacek, Gluba-Brzózka Anna, Franczyk Beata, Banach Maciej, Bartnicki Piotr
a Department of Nephrology, Hypertension and Family Medicine , Medical University of Lodz , Lodz , Poland.
c Healthy Aging Research Center , Medical University of Lodz , Lodz , Poland.
Expert Opin Pharmacother. 2016 Oct;17(15):2049-56. doi: 10.1080/14656566.2016.1232394. Epub 2016 Sep 21.
Despite progress in the understanding of pathogenetic mechanisms of organ cyst formation in autosomal dominant polycystic kidney disease, current treatment methods are insufficient. Experimental studies and clinical trials target at inhibition of cysts development and to slowing CKD progression.
The purpose of this analysis is to overview available literature regarding treatment of ADPKD. The most important recent events concerning ADPKD treatment are: the results of TEMPO 3/4 study and the registration of tolvaptan in the treatment of patients with CKD stage I-III and rapidly progressive ADPKD by EMA. ERA-EDTA recommendations for use of tolvaptan in ADPKD of 2016 will be useful for the identification of patients with rapid progression of disease who will benefit most from treatment. Clinical trials concerning inhibitors of mTOR and SSAs have not delivered convincing evidence of their effectiveness. Usefulness of statins in ADPKD require confirmation in adults. The HALT-PKD study confirmed that inhibition of RAA system slows progression of ADPKD.
Current treatment of ADPKD involves: the optimization of life style and combined pharmacological treatment with ACE inhibitors or angiotensin receptor blockers, statins (patients with lipid disorders and cardiovascular disease) and tolvaptan (patients with stage I-III CKD and rapidly progressive ADPKD).
尽管在常染色体显性多囊肾病中器官囊肿形成的发病机制理解方面取得了进展,但目前的治疗方法仍不充分。实验研究和临床试验旨在抑制囊肿发展并减缓慢性肾脏病进展。
本分析的目的是概述有关常染色体显性多囊肾病治疗的现有文献。常染色体显性多囊肾病治疗方面最近最重要的事件包括:TEMPO 3/4研究结果以及欧洲药品管理局批准托伐普坦用于治疗I-III期慢性肾脏病和快速进展性常染色体显性多囊肾病患者。2016年欧洲肾脏最佳实践(ERA-EDTA)关于在常染色体显性多囊肾病中使用托伐普坦的建议将有助于识别疾病快速进展且将从治疗中获益最大的患者。关于雷帕霉素靶蛋白(mTOR)抑制剂和生长抑素类似物(SSAs)的临床试验尚未提供其有效性的确凿证据。他汀类药物在常染色体显性多囊肾病中的有效性需要在成人中得到证实。HALT-PKD研究证实抑制肾素-血管紧张素-醛固酮系统(RAA系统)可减缓常染色体显性多囊肾病进展。
目前常染色体显性多囊肾病的治疗包括:优化生活方式以及联合使用血管紧张素转换酶(ACE)抑制剂或血管紧张素受体阻滞剂、他汀类药物(用于脂质紊乱和心血管疾病患者)和托伐普坦(用于I-III期慢性肾脏病和快速进展性常染色体显性多囊肾病患者)进行药物治疗。