A systematic review of the predictors of disease progression in patients with autosomal dominant polycystic kidney disease.

BACKGROUND: Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder characterised by progressive renal cyst formation leading to renal failure in the majority of patients. The likelihood and rate of ADPKD progression is difficult to predict and there is a clear need to identify prognostic indicators that could be used to anticipate ADPKD progression, to aid the management of patients in clinical practice. METHODS: A systematic literature review was conducted to identify publications detailing the natural history of ADPKD, including diagnosis, prognosis and progression. Publications were identified and filtered, and data were extracted, based on a predefined research protocol. RESULTS: The review identified 2799 journal articles and 444 conference abstracts; 254 articles, including observational studies, clinical trials and reviews, proceeded to data extraction. Disease progression was associated with a variety of prognostic indicators, most commonly age and total kidney volume (TKV). In the identified clinical trials, the absence of a consistent measure of disease progression led to variation in the primary endpoints used. Consequently, there was difficulty in consistently and effectively demonstrating and comparing the efficacy of investigational treatments across studies. More consistency was found in the observational studies, where disease progression was most frequently measured by TKV and glomerular filtration rate. CONCLUSIONS: This systematic review identified age and TKV as the most commonly cited prognostic indicators in the published ADPKD literature. It is envisaged that this review may inform future research, trial design and predictive models of ADPKD natural history, helping to optimise patient care.