PCR17 Utility Estimates for Treatment in Primary Hyperoxaluria Type 1

Primary hyperoxaluria type 1 (PH1) is a rare genetic disorder characterized by hepatic overproduction of oxalate. Excretion excess oxalate the kidneys leads to recurrent kidney stones, nephrocalcinosis, progressive disease, and multiorgan damage from systemic oxalosis. Patients progressing or presenting with failure require invasive approaches such as dialysis liver and/or transplantation. There currently little evidence about impact PH1 on health-related quality life (HRQoL). This study aimed understand burden patients eliciting utilities support future economic evaluations. A targeted literature review interviews one pediatric adult clinical expert (n=2) informed development health state vignettes describing PH1. states were defined based chronic disease (CKD) stages in adults children. post-combined transplant (CLKT) was also developed. The valued members UK general public using EQ-5D-5L time trade-off (TTO) methods. One hundred participants states; (50% male, mean age 41 years). Scores lowest for CKD5 child (EQ-5D-5L=-0.05, TTO=0.33) (EQ-5D-5L=0.02, TTO=0.39), large decrements observed between early CKD (CKD4 CKD5). CLKT scores comparable those (1-3b) both states. yielded novel set utility values Mean TTO ratings followed similar pattern, although consistently lower. results demonstrate considerable HRQoL associated PH1, this being greatest advances required. These data can be used evaluations treatments inform decision-making.