Utility of ultrasonography for predicting indications for tolvaptan in patients with autosomal dominant polycystic kidney disease

Purpose: Tolvaptan is the first approved treatment for autosomal dominant polycystic kidney disease (ADPKD) that targets a mechanism directly contributing to the development and growth of renal cysts. We investigated the ability of ultrasonography to predict total kidney volume (TKV) of 750 mL or more, which is an indication for tolvaptan therapy in patients with ADPKD.

Methods: A total of 46 patients with ADPKD were evaluated. The most statistically appropriate measurement based on ultrasonography for predicting TKV determined by computed tomography (CT) was assessed.

Results: TKV determined by CT was 796.8 (508.8-1,560.3) mL. The median length, anteroposterior distance, and mediolateral distance determined using ultrasonography were 15.7 cm, 7.6 cm, and 7.6 cm in the left kidney, and 13.4 cm, 6.9 cm, and 7.2 cm in the right kidney, respectively. Multivariate regression analysis showed that total kidney length (left and right) [variance inflation factor (VIF), 9.349] and total mediolateral distance (left and right) (VIF, 3.988) were independently associated with TKV. The correlation (r) between the logarithm of TKV determined by CT and total mediolateral distance determined using ultrasonography was 0.915 (p < 0.001). The linear regression equation was log (total kidney volume) = 1.833 + 0.075 × total mediolateral distance (left and right) based on ultrasonography. The area under the receiver operating characteristic curve for total mediolateral distance determined using ultrasonography to predict TKV of 750 mL or more was 0.989. Using the total mediolateral distance cut-off value of 14.2 cm, the sensitivity and specificity were 96.0% and 100.0%, respectively.

Conclusion: Total mediolateral distance determined using ultrasonography can predict TKV in patients with ADPKD.