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Published on: June 23, 2015
Pharmacologic Management of Autosomal Dominant Polycystic Kidney Disease
William Ackley1, Neera K Dahl2, Meyeon Park3
1Department of Nephrology, University of Connecticut, Nephrology, Farmington, CT.
Insights
Autosomal dominant polycystic kidney disease (ADPKD) is a common genetic kidney disorder. Tolvaptan is the only approved treatment targeting rapid progression, with other therapies also considered for ADPKD management.
Area of Science:
- Nephrology
- Genetics
- Pharmacology
Background:
- Autosomal dominant polycystic kidney disease (ADPKD) is the most frequent genetic kidney disorder.
- It is the fourth leading cause of end-stage kidney disease (ESKD).
- The pathogenesis of ADPKD is not fully understood, leading to significant morbidity beyond kidney disease.
Purpose of the Study:
- To detail the use and prescription of tolvaptan for ADPKD.
- To discuss other potential therapies for ADPKD treatment.
- To provide an overview of current ADPKD management strategies.
Main Methods:
- Review of current literature on ADPKD treatments.
- Analysis of tolvaptan's mechanism of action and clinical application.
- Discussion of emerging and supportive therapies for ADPKD.
Main Results:
- Tolvaptan, approved in 2018, is the sole mechanism-specific therapy for individuals at risk of rapid ADPKD progression.
- Risk assessment for rapid progression is a critical factor in treatment decisions.
- Various therapeutic approaches, including tolvaptan and others, are considered for managing ADPKD patients.
Conclusions:
- Tolvaptan represents a significant advancement in ADPKD management by targeting rapid progression.
- Comprehensive treatment strategies involve risk assessment and consideration of multiple therapeutic options.
- Further research into ADPKD pathogenesis may yield additional treatment targets.
Abstract:
Autosomal dominant polycystic kidney disease (ADPKD) is the most common genetic kidney disorder and the fourth leading cause of end-stage kidney disease. ADPKD encompasses a wide range of morbidity in addition to chronic kidney disease and end-stage kidney disease, and its pathogenesis remains incompletely understood. Progress in the management of this condition includes the 2018 FDA approval of tolvaptan as the only mechanism-specific treatment available for individuals at risk of rapid progression. Assessing the risk of rapid progression is discussed at greater length in a separate article in this special issue. This section will address use and prescription of tolvaptan in more detail and address other therapies that may be considered in the treatment of patients with ADPKD.
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