Abstract: FR-PO306

Efficacy and Safety of Tolvaptan in Autosomal Dominant Polycystic Kidney Disease with Renal Insufficiency

Session Information

Category: Genetic Diseases of the Kidney

  • 801 Cystic Kidney Diseases


  • Oguro, Masahiko, Toranomon Hospital, Kawasaki, Japan
  • Hoshino, Junichi, Toranomon Hospital, Kawasaki, Japan
  • Yamanouchi, Masayuki, Okinaka Memorial Institute for Medical Research, Tokyo, Japan
  • Ubara, Yoshifumi, None, Setagaya, TOKYO, Japan

A recent study demonstrated that tolvaptan slowed kidney volume growth and kidney function decline in autosomal dominant polycystic kidney disease (ADPKD) patients with creatinine clearance ≧60 ml per minute. However, tolvaptan’s efficacy in advanced chronic kidney disease (CKD) patients—especially those whose eGFR is <30ml per minute—has remained unknown.


In this prospective cohort study, 54 patients with ADPKD who had eGFR≧15 per minute and total kidney volume (TKV) ≧750 ml were treated with tolvaptan. The primary endpoint was a change in TKV and eGFR over 1 year treatment with tolvaptan across CKD stages G2-G4. The secondary endpoint was the final dose of tolvaptan and proportion of tolerance at each CKD stage.


The rate of kidney growth during the 1-year treatment didn’t differ significantly across CKD stages G2-G4. The median [IQR] of relative change in TKV in CKD stages G2, G3a, G3b, and G4 was, respectively, 5.70 [-0.67, 8.75], 6.66 [2.02, 17.66], 8.22 [5.97, 31.39], and 8.80 [4.12, 25.88] %/year (P=0.34). Nor did the rate of eGFR decline during the 1-year treatment differ significantly across CKD stages G2-G4. The relative annual change in eGFR—compared with the baseline eGFR—was, respectively, -4.15 [-11.78, 0.08], -7.28 [-11.17, -0.49], -7.02 [-16.74, -2.68], and -10.76 [-13.93, -18.6] %/year (P=0.59). Tolerance didn’t differ significantly across CKD stages G2-G4.


This analysis suggests that patients with advanced CKD stages—including G4—might benefit from treatment with tolvaptan as do patients with preserved kidney function.


  • Government Support - Non-U.S.