The natural history of
autosomal dominant polycystic kidney disease (
ADPKD) is characterized by a variable rate of
cyst development and increase in total kidney volume (TKV), variable kidney function decline and age of onset of
end-stage renal disease (
ESRD), and variable presentation of renal and extrarenal manifestations.
Precision medicine is proposed to improve patient outcomes by tailoring
therapy to the specific genetic background, pathophysiology, disease burden, prognosis and status of each individual. This approach to the management of patients with
ADPKD is nearing clinical implementation owing to advances in genetics, imaging,
biomarker development and
therapeutics. In this Review, we discuss pharmacological and non-pharmacological interventions for the treatment of
hypertension and
proteinuria, and for slowing the rate of
cyst growth in patients with
ADPKD before the development of
ESRD. We provide recommendations for the management of renal complications, including
cyst infection,
nephrolithiasis, haematuria and
chronic pain. The early treatment of patients with
ADPKD who are largely asymptomatic is associated with a therapeutic burden but slows
cyst growth and delays subsequent loss of kidney function, which ultimately delays the need for
renal replacement therapy and has a positive effect on the quality of life of patients.