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Use of Ultra-high Field MRI in Small Rodent Models of Polycystic Kidney Disease for In Vivo Phenotyping and Drug Monitoring
Published on: June 23, 2015
Treatment of Autosomal-Dominant Polycystic Kidney Disease
Sara S Jdiaa1, Reem A Mustafa2, Alan S L Yu2
1Division of Nephrology, University of Toronto, Toronto, ON, Canada.
Insights
Autosomal-dominant polycystic kidney disease (ADPKD) management focuses on identifying rapid progression risk. Current strategies include lifestyle changes, blood pressure control, and Tolvaptan, with new treatments emerging.
Area of Science:
- Nephrology
- Genetics
- Internal Medicine
Background:
- Autosomal-dominant polycystic kidney disease (ADPKD) is a leading cause of end-stage kidney disease affecting diverse populations.
- ADPKD presents with heterogeneous phenotypes, necessitating risk stratification for personalized therapeutic decisions.
- Predictive tools for ADPKD severity incorporate imaging, genetic, and clinical factors.
Purpose of the Study:
- To review current understanding and management strategies for ADPKD.
- To highlight tools for identifying patients at risk of rapid disease progression.
- To discuss emerging therapeutic interventions for ADPKD.
Main Methods:
- Review of existing literature and clinical trial data on ADPKD management.
- Analysis of tools for predicting ADPKD progression, including MRI-based kidney volume and eGFR.
- Evaluation of lifestyle modifications, blood pressure control, and pharmacologic treatments like Tolvaptan.
Main Results:
- Intensive blood pressure control, as supported by the HALT PKD trial, is crucial for patients with preserved kidney function.
- Lifestyle modifications such as weight management, salt restriction, and smoking cessation are beneficial.
- Tolvaptan is an effective disease-modifying agent for high-risk ADPKD patients, despite potential side effects.
Conclusions:
- Accurate identification of ADPKD patients at risk of rapid progression is key to guiding treatment.
- A multi-faceted approach combining lifestyle, blood pressure management, and targeted pharmacotherapy is essential.
- Ongoing research into novel interventions offers hope for improved ADPKD outcomes.
Abstract:
Autosomal-dominant polycystic kidney disease (ADPKD) is a chronic systemic disease that affects all races and ethnicities. It is the fourth leading cause of end-stage kidney disease, and it has a heterogenous phenotype ranging from mild to severe disease. Identifying patients with ADPKD who are at risk of rapid progression can guide therapeutic decisions. Several tools to predict disease severity are available, based on features such as total kidney volume assessed with magnetic resonance imaging, PKD genotype, estimated glomerular filtration rate (eGFR) trajectory, and the occurrence of hypertension and urologic complications early in life. During the past decade, more evidence has emerged regarding optimal ADPKD management. The HALT PKD (Halt Progression of Polycystic Kidney Disease) trial supported intensive blood pressure control in patients younger than 50 years of age with preserved kidney function. A healthy lifestyle, including maintaining a healthy weight, salt restriction, and smoking cessation, is likely to be beneficial. Tolvaptan, the only disease-modifying agent for patients with ADPKD at risk of rapid progression, is gaining wider use, but is still limited by its side effects. This is an exciting time for the ADPKD community because multiple promising interventions are in the pipeline and being investigated.
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