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Kidney Week

Abstract: TH-PO841

Two Years of Follow-Up Experience with Tolvaptan Treatment in Autosomal Dominant Polycystic Kidney Disease

Session Information

Category: Genetic Diseases of the Kidneys

  • 1001 Genetic Diseases of the Kidneys: Cystic


  • Martins Munoz, Judith Fatima, Hospital de Getafe, Madrid, Spain
  • Gutierrez-martinez, Eduardo, Hospital 12 de Octubre, Madrid, Spain
  • Ortiz, Alberto, Fundacion Jimenez Diaz, Madrid, Spain

Group or Team Name

  • Group of Polycystic Kidney Disease from Madrid

Autosomal dominant polycistic kidney disease (ADPKD) is the most common inherited kidney disease, it accounts for 10% of end renal disease cases. The aproval of tolvaptan in 2014 as the first targeted therapy of ADPKD was a significant progress. Since this year there are only a few data reported of the real experience with patients ADPKD. The objective of our study was to describe the effectiveness and safety profile of patients during the two first years of treatment.


Retrospective observacional study. We ncluded ADPKD patients with chronic kidney disease stage 1-3 and treated with Tolvaptan We collect data baseline data from 15 centres from Madrid (Spain) . These data inludes lab values and kidney volumen.


We included 143 patients. The mean follow up period was 8,7± 5,1 months. 51 % women, aged 42,7± 10,55 years with eGFR of 63,86±22,75 mL/min/1.73m2 and urine osmolality 445,89 ± 172,25 mOsm/kg. Baseline CKD: stage 1 (19,6%), 2 (34,3%) , 3a (27,3%), 3b (16,8%) and 4 (2,1%). The total kidney volumen (TKV) was 1696,47± 1399 and class of Mayo Clinic classification :1B (0,7%), 1C (23,8%), 1D (31,5%), 1E (24,5%). The medium dose achieved was 100,69 ± 6,54 mg in 4,9 ± 3,8 weeks .The rate of adverse events was 74,1%. 68,56%)were those related to increase aquaresis (thrist, polyuria ,nocturia and polidipsia) . A total of 14 cases (9,8 %) experienced an elevation of liver-enzyme levels,but no cases of sever liver injury was reported. 31 patients (21,6 %) discontinued treatment after a period of 10,9± 5,4 months.The main reasons to dicontinuate were: aquretic effects (86% %), elevations of liver-enzyme levels 7% and 3,5% other efects . 9 patients (29%) reinitaed the treatment after a medium period of 7,02 ± 4,11 months. Adherence to the teraphy was 84,6 %. Renal function (Efgr) at 1,6 ,12, 18 , 21 and 24 months was respectively:59, 83± 21,71 ml/min, 56,58 ± 20,22 ml/min, , 58,38±19,5 m/min, 60,21 ±21,85 and 62,87 ± 21,83.


In our experience Tolvaptan could be considered as a safety drug in patients with ADPKD. The adherence of the treatment was good, with similar data obtained in TEMPO 3:4 trial. Due to adverse events its neccesary monitor the enzyme liver levels, but no cases of fatal liver damage was reported. The renal function remains stable after two year of treatment.