Treatment of Autosomal Dominant Polycystic Kidney Disease: Integrating Clinical Practice Guidelines, Patient Perspectives, and Real-World Effectiveness.

Abstract

Autosomal dominant polycystic kidney disease (ADPKD) is a systemic, hereditary disorder requiring lifelong, multidisciplinary management. Approximately half of affected individuals progress to kidney failure by age 60. Recent advances in genetics, imaging, and disease pathophysiology have enabled earlier diagnosis, improved risk stratification, and led to the development of disease-modifying therapies (such as tolvaptan) for high-risk patients. Despite the availability of comprehensive clinical practice guidelines, real-world implementation is hindered by disease heterogeneity, psychosocial impacts, and the need for individualized care. The aim of this narrative review is to facilitate guideline implementation by integrating evidence from patient perspectives, real-world effectiveness, and offering strategies to overcome practical barriers.