Effect of statin therapy on disease progression in pediatric ADPKD: design and baseline characteristics of participants

Abstract

Autosomal dominant polycystic kidney disease (ADPKD) is the most common genetic kidney condition and is associated with important renal and cardiovascular manifestations in childhood. Renal cystic disease can be documented in some cases as early as in utero. Early intervention is critical if the long-term complications of this condition, including end-stage renal disease, are to be ameliorated. Here we describe our ongoing randomized double-blind placebo-controlled phase III clinical trial to assess the effect of pravastatin treatment on renal and cardiovascular disease progression in 107 children and young adults age 8-22 years with ADPKD who are receiving the angiotensin converting enzyme inhibitor lisinopril. Baseline demographic and laboratory data are provided. Results of this study could markedly impact the standard of care for evaluation and treatment of ADPKD in this population.

Figure 1

Representative image showing T1-weighted magnetic resonance image of kidneys with overlying grid. The analyst highlights the selected “+” points overlying the each kidney in each magnetic resonance image. This point count is converted to a pixel count to obtain the area measurements. The total kidney volume is then calculated from the set of contiguous images by summing the products of the area measurements and the slice thickness.

Figure 2

Magnetic resonance image showing highlighted renal cysts on T2-weighted image.