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BackgroundAutosomal dominant polycystic kidney disease (ADPKD), the leading monogenic cause of kidney failure, exhibits heterogeneous clinical progression. This systematic review and meta-analysis synthesize and evaluate current evidence on blood and urine prognostic biomarkers in ADPKD, addressing gaps in understanding their role in predicting progression and guiding clinical trial selection and management.MethodsWe searched PubMed, Embase, and Cochrane up to April 2025 and screened articles in duplicate. We included longitudinal studies evaluating the blood and urine prognostic biomarkers in patients with ADPKD with at least 10 participants and 1 year of follow-up. We used the Quality in Prognosis Studies tool to assess risk of bias, random effects meta-analyses to pool effect estimates, and the GRADE approach to assess the certainty of evidence.ResultsWe included 58 studies, with 33 urinary biomarkers and 29 serum/blood biomarkers identified. The most frequently studied biomarkers were urine osmolality, copeptin, proteinuria, Monocyte Chemoattractant Protein-1, and uric acid, whereas the most studied outcomes were estimated Glomerular Filtration Rate and Total Kidney Volume. The urinary biomarkers that showed the largest association with ADPKD were Monocyte Chemoattractant Protein-1, Kidney Injury Molecule-1, albumin, and Beta 2 microglobulin. Serum biomarkers associated with outcomes were primarily copeptin and Fibroblast Growth Factor-23, with β-Hydroxybutyrate and bicarbonate exhibiting lesser association.ConclusionIn conclusion, this systematic review highlights the potential prognostic value of blood and urine biomarkers in ADPKD. It also verified the need for further validation of biomarker use in ADPKD.Clinical trial numberNot applicable.

Abstract licence: CC BY-NC-ND