Two Year Experience with Tolvaptan in Patients with Rapidly Progressing Polycystic Kidney Disease

摘要

Background: Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder in which patients progress to end stage chronic kidney disease. Tolvaptan, vasopressin V2 receptor antagonist, has been postulated as an effective treatment to slow this progression. METHOD: All patients with ADPKD in whom treatment with tolvaptan was initiated were selected, with follow-up until 30 June 2019. The initial dose was 45/15 mg/day, with monthly titration at 60/30 mg and 90/30 mg. Monthly anthropometric, analytical and adverse effects were collected. RESULT: We present results from of 12 patients (mean age 39.8 +/- 7.3 years; 4 men and 8 women). 100% of the patients were selected to start tolvaptan because they had evidence of rapidly progressing disease, with a confirmed annual estimated filtration rate (eGFR) decline 5 mL/min/1.73m2 in 1 year, and/or 2.5 mL/min/1.73m2 per year over a period of 5 years. All patients had symptoms derived from aquaresis from the beginning of treatment. A decline in eGFR was observed in all patients after starting tolvaptan, dropping 38,8% from its baseline in one patient. In two patients the drug was temporarily discontinued due to hepatotoxicity, with subsequent recovery. Conclusion: Symptoms derived from aquaresis are very common and it is not known if they can limit the tolerability of the drug. An initial decline of the eGFR is observed during the follow-up. Close monitoring of liver function is important because of the potential hepatotoxicity of tolvaptan. More follow-up time is needed to asses the long-term efficacy and safety of tolvaptan.