Background and importance Tolvaptan is the first authorised drug for the treatment of autosomal dominant polycystic kidney disease (PQRAD).
Aim and objectives To analyse the efficacy and safety of tolvaptan in the treatment of PQRAD compared with the results of the TEMPO study.
Material and methods This was a descriptive, observational and retrospective study of patients treated with tolvaptan (August 2017–April 2019). Variables studied were: age, sex, arterial hypertension, total renal volume (VRT), creatinine, serum potassium and sodium, transaminases and glomerular filtration rate (GFR). Adverse reactions were recorded. For collection of data, the electronic medical history was used. Statistical analysis was performed with the Stata14 programme.
Results We included 23 patients (8 women, 5 men), median age 46 years (31–63) years. All had VRT >1000 mL (median 1920 mL (1230–3154)). At the beginning of treatment, GFR was 49.7 mL/min/1.73 m2 (25.6–102.31): 3 patients had stage 1 chronic kidney disease, 2 patients had stage 2, 10 patients stage 3A, 6 patients stage 3B and 2 patients stage 4. All patients suffered progressive deterioration of renal function during treatment: 5.25 mL/min/1.73m2 (−3.61–18.29) and 8.28 mL/min/1.73 m2 (−1.87–15.59) at 3 and 6 months, respectively, and 8.49 mL/min/1.73 m2 (4.21–14.06) at the end of the treatment year. Tolvaptan was suspended in three patients due to impaired renal function (GFR <20 mL/min/1.73 m2); all other patients were still receiving treatment at the end of the study (five with dose reduction to 60/30 mg). All patients reported polyuria and polydipsia and no patient suffered clinically relevant alterations in serum sodium or potassium. Relative to liver function, three patients suffered specific alterations in AST, ALT and GGT above normal values (57, 76 and 63 IU/L, respectively).
Conclusion and relevance Our results, compared with the TEMPO study, showed a higher rate of renal function deterioration, measured as a decrease in GFR rate after 1 year of treatment (8.49 vs 2.7 mL/min/1.73 m2), probably in relation to the worst baseline condition of the patients included in our study. Therefore, it is essential to identify the population susceptible to receiving this drug, prioritising those patients with GFR >45 mL/min/1.73 m2 and with a high risk of rapid progression.
References and/or acknowledgements TEMPO clinical trial.
No conflict of interest.
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