Prospective change in renal volume and function in children with ADPKD.

BACKGROUND AND OBJECTIVES Autosomal dominant polycystic kidney disease (ADPKD) is a progressive hereditary disorder affecting children and young adults. Early intervention may be necessary to significantly affect the long-term consequences of this disease. DESIGN, SETTING, PARTICIPANTS, & MEASUREMENTS The authors conducted a 5-yr randomized clinical trial to assess the effect of BP control with angiotensin-converting enzyme inhibition (ACEI) on disease progression in 85 children and young adults with ADPKD. Study groups were determined by subject BP, including hypertension (BP >or= 95th percentile), borderline hypertension (BP 75 to 95th percentile), and severe ADPKD (BP 10 renal cysts). The primary outcome variable was renal volume by ultrasound, with secondary outcome variables including left ventricular mass index (LVMI) and microalbuminuria. In secondary analysis, the authors compared results between hypertensive and normotensive groups. RESULTS The authors were not able to demonstrate a significant effect of ACEI on renal growth in young subjects with ADPKD. Hypertensive children were at particular risk for increases in renal volume and LVMI and decreased renal function as compared with the other study groups, and borderline hypertensive children were at high risk to develop hypertension over time. However, ACEI treatment was associated with stable renal function and LVMI in this group of children. CONCLUSIONS Close monitoring of cardiovascular and renal status is indicated in ADPKD children with hypertension or borderline hypertension. In contrast to effects in hypertensive ADPKD children, ACEI treatment in normotensive or borderline hypertensive ADPKD children may prevent the development of increased LVMI and deterioration in renal function.