Quality of life (QoL) for people with primary sclerosing cholangitis (PSC): a pragmatic strategy for identifying relevant QoL issues for rare disease.

ABSTRACT:

Background

Primary sclerosing cholangitis (PSC) is a rare incurable disease of the bile ducts and liver which can significantly impair quality of life (QoL). No existing QoL tools are entirely suitable for people living with PSC (PwPSC). We aimed to develop a measure of QoL for PwPSC in the UK, beginning by identifying relevant QoL issues. This paper describes our approach to this first stage, and discusses related benefits and limitations.

Methods

Scientific consensus on how to reliably stage PSC is lacking, due to its rarity and heterogeneity. We initially hypothesised four categories for PSC severity. After beginning the study, these were revised to six. For such a rare disease, the study could not recruit sufficient participants in each of these categories, particularly

SUBMITTER: Marcus E 

PROVIDER: S-EPMC9287498 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature