scholarly journals Clinical Trials in Pediatric Autosomal Dominant Polycystic Kidney Disease

2017 ◽  
Vol 5 ◽  
Author(s):  
Melissa A. Cadnapaphornchai
Keyword(s):  
Clinical Trials ◽  
Kidney Disease ◽  
Polycystic Kidney Disease ◽  
Autosomal Dominant ◽  
Polycystic Kidney ◽  
Autosomal Dominant Polycystic Kidney

scholarly journals Metabolic Reprogramming in Autosomal Dominant Polycystic Kidney Disease

2020 ◽  
Vol 15 (4) ◽  
pp. 577-584 ◽  
Author(s):  
Kristen L. Nowak ◽  
Katharina Hopp
Keyword(s):  
Clinical Trials ◽  
Kidney Disease ◽  
Polycystic Kidney Disease ◽  
Autosomal Dominant ◽  
Aerobic Glycolysis ◽  
Metabolic Reprogramming ◽  
Intermittent Fasting ◽  
Metabolic Demand ◽  
Polycystic Kidney ◽  
Autosomal Dominant Polycystic Kidney

Autosomal dominant polycystic kidney disease is characterized by progressive development and enlargement of kidney cysts, leading to ESKD. Because the kidneys are under high metabolic demand, it is not surprising that mounting evidence suggests that a metabolic defect exists in in vitro and animal models of autosomal dominant polycystic kidney disease, which likely contributes to cystic epithelial proliferation and subsequent cyst growth. Alterations include defective glucose metabolism (reprogramming to favor aerobic glycolysis), dysregulated lipid and amino acid metabolism, impaired autophagy, and mitochondrial dysfunction. Limited evidence supports that cellular kidney metabolism is also dysregulated in humans with autosomal dominant polycystic kidney disease. There are notable overlapping features and pathways among metabolism, obesity, and/or autosomal dominant polycystic kidney disease. Both dietary and pharmacologic-based strategies targeting metabolic abnormalities are being considered as therapies to slow autosomal dominant polycystic kidney disease progression and are attractive, particularly given the slowly progressive nature of the disease. Dietary strategies include daily caloric restriction, intermittent fasting, time-restricted feeding, a ketogenic diet, and 2-deoxy-glucose as well as alterations to nutrient availability. Pharmacologic-based strategies include AMP-activated kinase activators, sodium glucose cotransporter-2 inhibitors, niacinamide, and thiazolidenediones. The results from initial clinical trials targeting metabolism are upcoming and anxiously awaited within the scientific and polycystic kidney disease communities. There continues to be a need for additional mechanistic studies to better understand the role of dysregulated metabolism in autosomal dominant polycystic kidney disease and for subsequent translation to clinical trials. Beyond single-intervention trials focused on metabolic reprograming in autosomal dominant polycystic kidney disease, great potential also exists by combining metabolic-focused therapeutic approaches with compounds targeting other signaling cascades altered in autosomal dominant polycystic kidney disease, such as tolvaptan.

Clinical Trials in Autosomal Dominant Polycystic Kidney Disease

2015 ◽  
pp. 109-135 ◽  
Author(s):  
Alan S.L. Yu ◽  
◽  
Mireille El-Ters ◽  
Franz T. Winklhofer ◽  
◽  
...  
Keyword(s):  
Clinical Trials ◽  
Kidney Disease ◽  
Polycystic Kidney Disease ◽  
Autosomal Dominant ◽  
Polycystic Kidney ◽  
Autosomal Dominant Polycystic Kidney

scholarly journals Standardizing total kidney volume measurements for clinical trials of autosomal dominant polycystic kidney disease

2018 ◽  
Vol 12 (1) ◽  
pp. 71-77 ◽  
Author(s):  
Marie E Edwards ◽  
Jaime D Blais ◽  
Frank S Czerwiec ◽  
Bradley J Erickson ◽  
Vicente E Torres ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Kidney Disease ◽  
Polycystic Kidney Disease ◽  
Autosomal Dominant ◽  
Kidney Volume ◽  
Polycystic Kidney ◽  
Total Kidney Volume ◽  
Volume Measurements ◽  
Autosomal Dominant Polycystic Kidney
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