Triptolide-containing formulation in patients with autosomal dominant polycystic kidney disease and proteinuria: An uncontrolled trial

摘要

Autosomal dominant polycystic kidney disease (ADPKD) accounts for 5%-10% of patients with ESRD, making it the most common inherited cystic renal disease. Overt proteinuria is unusual in ADPKD (<25% of patients have levels > 300 mg/d), but proteinuria is a risk factor for rapid cyst growth and the development of ESRD. Based on a limited number of biopsies in ADPKD patients with heavy proteinuria, the most common underlying pathologic changes are focal segmental glomerulo-sclerosis, membranous nephropathy, and minimal change disease. The traditional Chinese medicine Tripterygium wilfordii (also called thunder god vine or Lei Gong Teng) contains triptolide and has been used widely as an effective antiproteinuric. Tripterygium has an impressive antiproteinuric effect in various forms of CKD, including the pathologic lesions mentioned. Recently, triptolide was shown in experimental studies to inhibit cyst formation and growth. In this uncontrolled trial, we treated 9 proteinuric ADPKD patients with Tripterygium to test for antiproteinuric and/ or cyst-inhibitory effects.