PCR17 Utility Estimates for Treatment in Primary Hyperoxaluria Type 1

Abstract

Primary hyperoxaluria type 1 (PH1) is a rare genetic disorder characterized by hepatic overproduction of oxalate. Excretion of excess oxalate by the kidneys leads to recurrent kidney stones, nephrocalcinosis, progressive kidney disease, and multiorgan damage from systemic oxalosis. Patients progressing to or presenting with kidney failure require invasive approaches such as dialysis and liver and/or kidney transplantation. There is currently little evidence about the impact of PH1 on health-related quality of life (HRQoL). This study aimed to understand the burden of PH1 on patients by eliciting utilities to support future economic evaluations.