常染色体显性多囊肾病(autosomal dominant polycystic kidney disease,ADPKD)是人类最常见的单基因遗传性肾脏疾病,其特点是双肾无数囊肿形成并进行性增大,破坏肾脏正常结构和功能,最后进展至终末期肾脏疾病。目前临床上尚无有效抑制囊肿形成和增大的治疗措施。雷公藤内酯醇(triptolide,TL)是从中药雷公藤中提取的环氧二萜类化合物,具有抗炎、抗增殖等多种作用,多个动物实验研究显示其具有抑制囊肿形成以及保护肾功能的作用,为将来的临床应用奠定了基础。
Autosomal dominant polycystic kidney disease(ADPKD) is the most common single gene hereditary kidney disease,which is characterized by formation and persistent growth of renal cysts,damaging the structure and function of the kidneys and finally leading to end stage renal disease.By now there has been no effective therapy in clinical practice.Triptolide,an active diterpene extracted from a traditional Chinese medicine Leigongteng,has anti-inflammation and anti-proliferation effect.Animal studies have shown that triptolide can inhibit cyst formation and protect the renal function,laying a foundation for future clinical use.