Abstract
BackgroundTolvaptan is a vasopressin type 2 receptor antagonist and has been used to treat autosomal dominant polycystic kidney disease (ADPKD) since 2014. There has been limited real-world data on the safety and efficacy of tolvaptan.MethodsThis post-marketing surveillance was conducted to evaluate the long-term safety and the efficacy of tolvaptan in Japanese patients with ADPKD in real-world clinical settings. The baseline characteristics of 1630 patients treated with tolvaptan are reported. Safety analysis comprises evaluation of adverse drug reactions (ADRs). The efficacy evaluation includes percent change in total kidney volume (TKV) and change in estimated glomerular filtration rate (eGFR) before and after tolvaptan treatment.ResultsMean age was 49.7 ± 11.2 years and 843 (51.7%) patients were male. Baseline TKV was 2158 ± 1346 mL and eGFR was 44.4 ± 21.7 mL/min/1.73 m2. The majority of CKD patients were stage G3b (27.0%) and G4 (30.1%). Frequently reported ADRs were hepatic function abnormal (8.3%), thirst (8.2%), and hyperuricaemia (6.9%). The frequency of ALT elevation (> 30 and > 90 IU/L) was slightly high (32.9 and 8.3%) to previous studies. After tolvaptan treatment, the annual rate of percentage change in TKV reduced from 11.68%/year to 2.73%/year (P < 0.0001). Similar results were also obtained for the effect on change in eGFR from − 3.31 to − 2.28 mL/min/1.73 m2/year after initiation of tolvaptan treatment (P = 0.0403).ConclusionThere were no major problems with safety of tolvaptan treatment and comparable efficacy for TKV and eGFR was observed in relation to the previous pivotal two randomized control trials in this post-marketing surveillance.
Highlights
Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary monogenic kidney disorder and the fourth leading cause of end-stage kidney disease (ESKD) in adults worldwide [1, 2]
The baseline characteristics of 1630 patients treated with tolvaptan for the first time are reported; a further 78 patients were part of the phase 3b TEMPO extension study, the participation status of the remaining 11 patients in the TEMPO study is not known
We analyzed the safety and efficacy of tolvaptan in real-world settings based on the post-marketing surveillance (PMS) database containing data from March 2014 to May 2019
Summary
Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary monogenic kidney disorder and the fourth leading cause of end-stage kidney disease (ESKD) in adults worldwide [1, 2] It is a heterogeneous disorder resulting from mutations in two genes, PKD 1 and PKD 2 [1, 2]. Low intracellular calcium triggers increased adenosine-3’, 5’-cyclic monophosphate (cAMP) levels This plays a major role in cyst development and contributes to the development of ADPKD. Methods This post-marketing surveillance was conducted to evaluate the long-term safety and the efficacy of tolvaptan in Japanese patients with ADPKD in real-world clinical settings. Conclusion There were no major problems with safety of tolvaptan treatment and comparable efficacy for TKV and eGFR was observed in relation to the previous pivotal two randomized control trials in this post-marketing surveillance
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