Quality of life in primary sclerosing cholangitis: a systematic review

Abstract

Background Primary sclerosing cholangitis (PSC) is a rare bile duct and liver disease which can considerably impact quality of life (QoL). As part of a project developing a measure of QoL for people with PSC, we conducted a systematic review with four review questions. The first of these questions overlaps with a recently published systematic review, so this paper reports on the last three of our initial four questions: (A) How does QoL in PSC compare with other groups?, (B) Which attributes/factors are associated with impaired QoL in PSC?, (C) Which interventions are effective in improving QoL in people with PSC?. Methods We systematically searched five databases from inception to 1 November 2020 and assessed the methodological quality of included studies using standard checklists. Results We identified 28 studies: 17 for (A), ten for (B), and nine for (C). Limited evidence was found for all review questions, with few studies included in each comparison, and small sample sizes. The limited evidence available indicated poorer QoL for people with PSC compared with healthy controls, but findings were mixed for comparisons with the general population. QoL outcomes in PSC were comparable to other chronic conditions. Itch, pain, jaundice, severity of inflammatory bowel disease, liver cirrhosis, and large-duct PSC were all associated with impaired QoL. No associations were found between QoL and PSC severity measured with surrogate markers of disease progression or one of three prognostic scoring systems. No interventions were found to improve QoL outcomes. Conclusion The limited findings from included studies suggest that markers of disease progression used in clinical trials may not reflect the experiences of people with PSC. This highlights the importance for clinical research studies to assess QoL alongside clinical and laboratory-based outcomes. A valid and responsive PSC-specific measure of QoL, to adequately capture all issues of importance to people with PSC, would therefore be helpful for clinical research studies.

Health‐related quality of life in patients with primary sclerosing cholangitis: A longitudinal population‐based cohort study

“…1 The disease burden that is associated with PSC is thought to have a significant effect on health-related quality of life (HRQoL), although only limited evidence is available. 3 Proper estimation of HRQoL of PSC patients is needed for various purposes such as disease burden estimation, treatment evaluation in daily clinical practice and clinical trials, and for health-economic evaluation of new interventions. Both the US Food and Drug Administration and the European Medicines Agency have stressed the importance of HRQoL as an outcome measure in chronic diseases such as PSC.…”

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

“…It is important to include patient-reported outcomes (PROs), such as QoL, in clinical research to ensure that the safety and efficacy of new treatments is assessed holistically [ 9 ]. Our recent review found very few PSC clinical trials which assessed QoL [ 10 ]. In our review we also observed that generic QoL questionnaires may lack sensitivity and fail to measure important impacts of PSC, such as fatigue, and that surrogate endpoints commonly used in trial settings (e.g.…”