Research & Literature
Explore the leading researchers and institutions driving advances in this area, and dive into the full body of literature that informs this resource.
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Mayo Clinic
Rochester, United States
Broad Institute
Cambridge, United States
University Medical Center Groningen
Groningen, The Netherlands
National Institutes of Health
Bethesda, United States
Mayo Clinic in Florida
Jacksonville, United States
BGI Group (China)
Shenzhen, China
Yale University
New Haven, United States
Balıkesir University
Balıkesir, Türkiye
MACOM (United States)
Lowell, United States
KU Leuven
Leuven, Belgium
References
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Multicenter, open-label, extension trial to evaluate the long-term efficacy and safety of early versus delayed treatment with tolvaptan in autosomal dominant polycystic kidney disease: the TEMPO 4:4 Trial.
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[Quality of life in patients with autosomal dominant polycystic kidney disease].
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Nephrologie & therapeutique 2017; (13(7)):505-510 doi:10.1016/j.nephro.2016.12.005.
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New treatment paradigms for ADPKD: moving towards precision medicine.
Lanktree MB, Chapman AB
Nature reviews. Nephrology 2017; (13(12)):750-768 doi:10.1038/nrneph.2017.127.
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Can we further enrich autosomal dominant polycystic kidney disease clinical trials for rapidly progressive patients? Application of the PROPKD score in the TEMPO trial.
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Prognostic Enrichment Design in Clinical Trials for Autosomal Dominant Polycystic Kidney Disease: The TEMPO 3:4 Clinical Trial.
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Patterns of Kidney Function Decline in Autosomal Dominant Polycystic Kidney Disease: A Post Hoc Analysis From the HALT-PKD Trials.
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Burden of Proof for Tolvaptan in ADPKD: Did REPRISE Provide the Answer?
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A Practical Guide for Treatment of Rapidly Progressive ADPKD with Tolvaptan.
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Autosomal dominant PKD gets an atomic map.
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Journal of magnetic resonance imaging : JMRI 2019; (50(1)):41-51 doi:10.1002/jmri.26627.
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A rapid high-performance semi-automated tool to measure total kidney volume from MRI in autosomal dominant polycystic kidney disease.
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MR Angiography Screening and Surveillance for Intracranial Aneurysms in Autosomal Dominant Polycystic Kidney Disease: A Cost-effectiveness Analysis.
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Anterior Cerebral Artery Dissection in a Patient With Autosomal Dominant Polycystic Kidney Disease.
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Native Nephrectomy in Patients With Autosomal Dominant Polycystic Kidney Disease Evaluated for Kidney Transplantation.
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Metabolic Reprogramming in Autosomal Dominant Polycystic Kidney Disease: Evidence and Therapeutic Potential.
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Clinical journal of the American Society of Nephrology : CJASN 2020; (15(4)):577-584 doi:10.2215/CJN.13291019.
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Copy Number Variation: A New Genetic Form of Polycystic Kidney and Liver Disease.
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Gene Panel Analysis in a Large Cohort of Patients With Autosomal Dominant Polycystic Kidney Disease Allows the Identification of 80 Potentially Causative Novel Variants and the Characterization of a Complex Genetic Architecture in a Subset of Families.
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Management of Autosomal Dominant Polycystic Kidney Disease (ADPKD) During Pregnancy: Risks and Challenges.
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Insights into Autosomal Dominant Polycystic Kidney Disease from Genetic Studies.
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Hemorrhagic Cysts and Other MR Biomarkers for Predicting Renal Dysfunction Progression in Autosomal Dominant Polycystic Kidney Disease.
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Biallelic inheritance of hypomorphic PKD1 variants is highly prevalent in very early onset polycystic kidney disease.
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[Cardiovascular disorders in autosomal dominant polycystic kidney disease].
Hamzaoui M, Lamy G, Bellien J, Guerrot D
Nephrologie & therapeutique 2021; (17(1)):18-29 doi:10.1016/j.nephro.2020.09.003.
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Assessing Risk of Rapid Progression in Autosomal Dominant Polycystic Kidney Disease and Special Considerations for Disease-Modifying Therapy.
Chebib FT, Torres VE
American journal of kidney diseases : the official journal of the National Kidney Foundation 2021; (78(2)):282-292 doi:10.1053/j.ajkd.2020.12.020.
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Regulatory guidelines do not accurately predict tolvaptan and metabolite interactions at BCRP, OATP1B1, and OAT3 transporters.
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Clinical and translational science 2021; (14(4)):1535-1542 doi:10.1111/cts.13017.
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Effect of tolvaptan in Japanese patients with autosomal dominant polycystic kidney disease: a post hoc analysis of TEMPO 3:4 and TEMPO Extension Japan.
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Safety Profile of Tolvaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease.
Bellos I
Therapeutics and clinical risk management 2021; (17()):649-656 doi:10.2147/TCRM.S286952.
PMID: 34234441 - 56
What are the information needs and concerns of individuals with Polycystic Kidney Disease? Results of an online survey using Facebook and social listening analysis.
Ma T, Lambert K
BMC nephrology 2021; (22(1)):263 doi:10.1186/s12882-021-02472-1.
PMID: 34261447 - 57
Birth of two healthy girls following preimplantation genetic diagnosis and gestational surrogacy in a rapidly progressive autosomal dominant polycystic kidney disease case using tolvaptan.
Peces R, Mena R, Peces C, et al.
Clinical kidney journal 2021; (14(8)):1987-1989 doi:10.1093/ckj/sfab082.
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Predicting Future Renal Function Decline in Patients with Autosomal Dominant Polycystic Kidney Disease Using Mayo Clinic Classification.
Borrego Utiel FJ, Esteban de la Rosa RJ, Merino García E, et al.
American journal of nephrology 2021; (52(8)):630-641 doi:10.1159/000518255.
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Detection of PKD1 and PKD2 Somatic Variants in Autosomal Dominant Polycystic Kidney Cyst Epithelial Cells by Whole-Genome Sequencing.
Zhang Z, Bai H, Blumenfeld J, et al.
Journal of the American Society of Nephrology : JASN 2021; (32(12)):3114-3129 doi:10.1681/ASN.2021050690.
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Mayo imaging classification is a good predictor of rapid progress among Korean patients with autosomal dominant polycystic kidney disease: results from the KNOW-CKD study.
Park HC, Hong Y, Yeon JH, et al.
Kidney research and clinical practice 2022; (41(4)):432-441 doi:10.23876/j.krcp.21.261.
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Raynaud's phenomenon triggered by the vasopressin V2 receptor antagonist tolvaptan in a patient with autosomal dominant polycystic kidney disease and Sjögren's syndrome.
Roca Oporto FJ, Rocha JL
Clinical kidney journal 2022; (15(4)):827-828 doi:10.1093/ckj/sfab260.
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Cilia-Localized Counterregulatory Signals as Drivers of Renal Cystogenesis.
Walker RV, Maranto A, Palicharla VR, et al.
Frontiers in molecular biosciences 2022; (9()):936070 doi:10.3389/fmolb.2022.936070.
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Clinical Utility and Tolerability of Tolvaptan in the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD).
Raina R, Houry A, Rath P, et al.
Drug, healthcare and patient safety 2022; (14()):147-159 doi:10.2147/DHPS.S338050.
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The inhibition of MDM2 slows cell proliferation and activates apoptosis in ADPKD cell lines.
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Biology of the cell 2023; (115(1)):e2200037 doi:10.1111/boc.202200037.
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A disease progression model estimating the benefit of tolvaptan on time to end-stage renal disease for patients with rapidly progressing autosomal dominant polycystic kidney disease.
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BMC nephrology 2022; (23(1)):334 doi:10.1186/s12882-022-02956-8.
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Echocardiographic Abnormalities in Autosomal Dominant Polycystic Kidney Disease (ADPKD) Patients.
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Cystic Kidney Diseases That Require a Differential Diagnosis from Autosomal Dominant Polycystic Kidney Disease (ADPKD).
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Journal of clinical medicine 2022; (11(21)) doi:10.3390/jcm11216528.
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Autosomal Dominant Polycystic Kidney Disease: Role of Imaging in Diagnosis and Management.
Odedra D, Sabongui S, Khalili K, et al.
Radiographics : a review publication of the Radiological Society of North America, Inc 2023; (43(1)):e220126 doi:10.1148/rg.220126.
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Atypical Polycystic Kidney Disease as defined by Imaging.
Iliuta IA, Win AZ, Lanktree MB, et al.
Scientific reports 2023; (13(1)):2952 doi:10.1038/s41598-022-24104-w.
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Cardiac Manifestations in Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD): A Single-Center Study.
Arjune S, Grundmann F, Todorova P, et al.
Kidney360 2023; (4(2)):150-161 doi:10.34067/KID.0002942022.
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Mutation Analysis of Autosomal-Dominant Polycystic Kidney Disease Patients.
Suzuki Y, Katayama K, Saiki R, et al.
Genes 2023; (14(2)) doi:10.3390/genes14020443.
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Nonpharmacological Management of Autosomal Dominant Polycystic Kidney Disease.
Steele CN, Nowak KL
Advances in kidney disease and health 2023; (30(3)):220-227 doi:10.1053/j.akdh.2022.12.008.
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Effects of tolvaptan discontinuation in patients with autosomal dominant polycystic kidney disease: a post hoc pooled analysis.
Lioudis M, Zhou X, Davenport E, et al.
BMC nephrology 2023; (24(1)):182 doi:10.1186/s12882-023-03247-6.
PMID: 37349694 - 74
Many lessons still to learn about autosomal dominant polycystic kidney disease.
Orr S, Sayer JA
Journal of rare diseases (Berlin, Germany) 2023; (2(1)):13 doi:10.1007/s44162-023-00017-8.
PMID: 37664187 - 75
Case report: Genotype-phenotype characteristics of nine novel PKD1 mutations in eight Chinese patients with autosomal dominant polycystic kidney disease.
Zhuang J, Aierken A, Yalikun D, et al.
Frontiers in medicine 2023; (10()):1268307 doi:10.3389/fmed.2023.1268307.
PMID: 37901409 - 76
Assessing the Risk of Progression to Kidney Failure in Patients With Autosomal Dominant Polycystic Kidney Disease.
Lanktree MB, Kline T, Pei Y
Advances in kidney disease and health 2023; (30(5)):407-416 doi:10.1053/j.akdh.2023.06.002.
PMID: 38097331 - 77
Real-life use of tolvaptan in ADPKD: a retrospective analysis of a large Canadian cohort.
Calvaruso L, Yau K, Akbari P, et al.
Scientific reports 2023; (13(1)):22257 doi:10.1038/s41598-023-48638-9.
PMID: 38097698 - 78
Patient-Reported Outcomes Measures, Polycystic Kidney Disease Burden, and Outcomes in Autosomal Dominant Polycystic Kidney Disease.
Oberdhan D, Yarlas A, Bjorner JB, Krasa H
Kidney medicine 2024; (6(1)):100755 doi:10.1016/j.xkme.2023.100755.
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Validation of the Mayo Imaging Classification System for Predicting Kidney Outcomes in ADPKD.
Bais T, Geertsema P, Knol MGE, et al.
Clinical journal of the American Society of Nephrology : CJASN 2024; (19(5)):591-601 doi:10.2215/CJN.0000000000000427.
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Comprehensive Analysis of PKD1 and PKD2 by Long-Read Sequencing in Autosomal Dominant Polycystic Kidney Disease.
Xu D, Mao A, Chen L, et al.
Clinical chemistry 2024; (70(6)):841-854 doi:10.1093/clinchem/hvae030.
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Clinical Characteristics and Kidney Outcomes in Chinese Patients with Autosomal Dominant Polycystic Kidney Disease.
Fung WW, Szeto CC, Chow KM, et al.
Kidney360 2024; (5(5)):715-723 doi:10.34067/KID.0000000000000433.
PMID: 38556647 - 82
Predictors of autosomal dominant polycystic kidney disease progression: a Brazilian single-center cohort.
Nishimoto IH, Santos AG, Bianchini JM, et al.
Jornal brasileiro de nefrologia 2024; (46(3)):e20230040 doi:10.1590/2175-8239-JBN-2023-0040en.
PMID: 38935976 - 83
Mental Health and Autosomal Dominant Polycystic Kidney Disease: A Narrative Review.
Ebrahimi N, Garimella PS, Chebib FT, et al.
Kidney360 2024; (5(8)):1200-1206 doi:10.34067/KID.0000000000000504.
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RETRACTED: Determinants of Disease Progression in Autosomal Dominant Polycystic Kidney Disease.
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Journal of personalized medicine 2024; (14(9)) doi:10.3390/jpm14090936.
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Treatment of Autosomal-Dominant Polycystic Kidney Disease.
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American journal of kidney diseases : the official journal of the National Kidney Foundation 2025; (85(4)):491-500 doi:10.1053/j.ajkd.2024.08.008.
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Risk factors for unruptured intracranial aneurysms in asymptomatic patients with autosomal dominant polycystic kidney disease: who needs screening? A systematic review and meta-analysis.
Nguyen BA, Halpin B, Olson V, et al.
Journal of neurosurgery 2025; (143(1)):220-231 doi:10.3171/2024.9.JNS241175.
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Overweight Status, Obesity, and Progression to ESKD in Patients with Autosomal Dominant Polycystic Kidney Disease.
Nowak KL, Copeland TP, Ku E, et al.
Clinical journal of the American Society of Nephrology : CJASN 2025; (20(4)):520-528 doi:10.2215/CJN.0000000640.
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Autosomal Dominant Polycystic Kidney Disease: A Review.
Chebib FT, Hanna C, Harris PC, et al.
JAMA 2025; (333(19)):1708-1719 doi:10.1001/jama.2025.0310.
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Overview of ADPKD in Pregnancy.
Campbell RE, Edelstein CL, Chonchol M
Kidney international reports 2025; (10(4)):1011-1019 doi:10.1016/j.ekir.2024.12.035.
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