Safety and Tolerability of Tolvaptan in an Autosomal Dominant Polycystic Kidney Disease Spanish Cohort: A Real-World Experience.

Xavier E Guerra-Torres,J Peña Esparragoza,M Fernandez Rodríguez,J Mancha Ramos,M Perez Fernandez,P Martinez Miguel,D Rodriguez Puyol,H Bouarich

doi:10.7759/cureus.10207

Abstract

Introduction: Autosomal dominant polycystic kidney disease (ADPKD) is the commonest inherited disorder of the kidneys. A vasopressin V2-receptor antagonist (tolvaptan) was recently approved for the treatment of ADPKD. This study aims to analyze the safety and tolerability of tolvaptan for the management of ADPKD patients in a real-world setting.Methods: We conducted a descriptive retrospective study in ADPKD patients in an outpatient clinic setting in Spain from 2018 to 2019. Descriptive statistical analysis of demographics and clinical data, at baseline and one year after tolvaptan initiation, was assessed. Data are presented as median and interquartile range, and as frequencies for categorical variables.Results: Ten patients with ADPKD were identified. At baseline median age was 49.5 (38.5-63.5) years and 60% were males. During treatment with tolvaptan, no significant aquaresis-related symptoms or hepatotoxicity were described. No serious adverse events, discontinuation, or deaths were reported during the study.Conclusion: Tolvaptan was well-tolerated without severe adverse events in patients with ADPKD who showed rapid disease progression criteria. Longer follow-up is required to learn about the long-term effects of this treatment.

Highlights

Autosomal dominant polycystic kidney disease (ADPKD) is the commonest inherited disorder of the kidneys
ADPKD is characterized by mutations in polycystic kidney disease 1 (PKD1), polycystic kidney disease 2 (PKD2) and glucosidase II alpha subunit (GANAB) genes that increase the cyclic adenosine monophosphate intracellular levels and upregulate the mammalian target of rapamycin receptor
Tolvaptan showed a reduction in kidney volume, decrease in the slope of serum creatinine and glomerular filtration rate worsening while reducing the frequency of ADPKD-related complications derived from large kidney volume

Summary

Introduction

Autosomal dominant polycystic kidney disease (ADPKD) is the commonest inherited disorder of the kidneys. Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited disorder of the kidneys. ADPKD is characterized by mutations in polycystic kidney disease 1 (PKD1), polycystic kidney disease 2 (PKD2) and glucosidase II alpha subunit (GANAB) genes that increase the cyclic adenosine monophosphate (cAMP) intracellular levels and upregulate the mammalian target of rapamycin receptor (mTOR). These abnormalities result in the development and enlargement of kidney cysts and cell proliferation, leading to ESRD. Patients with ADPKD could have safety or tolerability concerns like increased thirst, concurrent comorbidities, major side effects leading to non-compliance with therapies, or taking therapies likely to interfere with tolvaptan

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