MO025: Long-term follow-up of patients with autosomal dominant polycystic kidney disease treated with tolvaptan

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Abstract BACKGROUND AND AIMS Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited renal disease characterized by the formation of cysts that destroy normal renal parenchyma. Tolvaptan (vasopressin antagonist) is the only drug approved today to slow disease progression. To assess the efficacy and tolerability of long-term treatment in patients with ADPKD. METHOD Retrospective observational study of ADPKD patients on tolvaptan treatment after its approval in Spain in 2017. RESULTS Of the 182 patients with ADPKD in follow-up in our practice, 18% (n = 33) initiated treatment because they met criteria for rapid progression. A total of 60.6% (n = 20) males with age 47.64 ± 8.64 years, a baseline GFR of 65.24 ± 20.14 mL/min and a height-adjusted total kidney volume (TKV) of 1137.45 ± 667.58 mL. A total of 72.7% reached the maximum dose (120 mg) with a mean maximum dose of 108.18 ± 22.42 mg and a mean time to reach it of 5.8 ± 9.3 weeks. A total of 90.9% (n = 33) presented side effects: 81.8% (n = 27) aquaretic and 12.1% (n = 4) hepatotoxicity. Other side effects observed were as follows: hypernatremia 9.1% (n = 3), hyperuricemia in 24% (n = 8). No other side effects were described. A total of 39.4% (n = 13) of the patients temporarily abandoned treatment after a mean time of 3.01 ± 2.17 months. The reasons for discontinuation were as follows: aquaretic effects 35.7% (n = 5), hepatotoxicity 35.7% (n = 5), 3.03% desire for pregnancy (n = 1), 3.03% cerebral hemorrhage (n = 1) and 3.03 (n = 1) worsening of renal function. Some 30.76% (n = 4) of the patients restarted treatment at 5.5 ± 6.76 months. The reason for previous withdrawal was hepatotoxicity (n = 2) and aquaretic effects (n = 2). Therefore, the definitive abandonment of treatment was 27.2%. The mean follow-up time of the patients was 39.85 ± 10.04 months. CONCLUSION Long-term use of tolvaptan in patients with ADPKD can be considered safe both from the point of view of renal function and side effects. The aquaretic effects are the most frequent. The aquaretic effects and hepatotoxicity are the main reasons for treatment discontinuation. All cases of hepatotoxicity reverted to normal, thus not being considered a serious effect in our experience.