Quantifying the impact of symptomatic acute hepatic porphyria on well-being via patient-reported outcomes: Results from the Porphyria Worldwide Patient Experience Research (POWER) study.

ABSTRACT: Acute hepatic porphyria (AHP) is a group of rare genetic diseases of heme biosynthesis resulting in severe neurovisceral attacks and chronic complications that negatively impact patients' well-being. This study evaluated the impacts of AHP on patients' physical and emotional health from a global perspective. Adult patients from the United States, Italy, Spain, Australia, Mexico, and Brazil with AHP with >1 porphyria attack within the past 2 years or receiving intravenous hemin and/or glucose for attack prevention completed an online survey assessing demographics, health characteristics, and patient-reported outcomes. Results were analyzed collectively and by patient subgroups. Ninety-two patients with AHP across the six countries completed the survey. More than 70% of patients reported tha

SUBMITTER: Dickey A 

PROVIDER: S-EPMC9830021 | biostudies-literature | 2023 Jan

REPOSITORIES: biostudies-literature