Editorial: Patient Reported Outcomes Provide Insight Into Disease Severity for People Living With Primary Sclerosing Cholangitis

Abstract

Primary sclerosing cholangitis (PSC) is a rare autoimmune liver disease (AILD) of unknown aetiology. It is a progressive disease that leads to cirrhosis and liver failure with no curative therapy other than liver transplantation [1]. The physical burden and uncertainty associated with PSC have a significant impact on patient quality of life (QOL) [2, 3]. However, insufficient studies have investigated this [4, 5]. Tan et al. sought to characterise the impact of the disease on QOL in PSC patients living in Australia and investigate the relationship between demographics, disease, and patient reported outcomes (PROs).

The group conducted a prospective observational cohort study with patients from nine tertiary liver centres in Australia. In total, 55 adult patients with PSC were recruited. Each participant was provided with a baseline PSC QOL questionnaire. Those with concomitant IBD also completed an IBD questionnaire. Clinical data was collected for all patients [6] (Figure 1).

FIGURE 1

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Patient reported symptoms (created in https://BioRender.com).

Results showed that fatigue was the most commonly reported symptom. Overall scores for the PSC PRO were low and IBD questionnaire high, indicating disease did not have a significant impact on QOL in this cohort. A positive correlation was found between PSC and IBD symptoms in patients. Decompensated cirrhosis and a history of cholangitis were associated with lower QOL. This is the first study to find an association with the latter [6]. Unlike in previous studies, older age was protective, and both sex and PSC phenotype were not associated with QOL [4-6]. However, the fact that concomitant IBD did not affect QOL, while cirrhosis was found to have a negative impact, is in line with previous work [4, 5].

Several limitations affect this study's generalisability. To begin with, the small cohort mainly consists of patients with less severe disease. In addition, patients from tertiary centers typically differ in their clinical characteristics when compared to other patients [7]. The study also strictly enrolled English-speaking individuals, meaning results do not represent the experience of marginalised groups. The cross-sectional nature of this study also fails to account for the variable experience of PSC.

Future research should focus on collecting longitudinal data from larger, diverse, prospective cohorts. Studies could administer additional questionnaires that look at specific symptoms (e.g., fatigue) and allow for comparison of QOL scores (e.g., Chronic Liver Disease Questionnaire (CLDQ)). Having a control group of patients with a similar disease (e.g., PBC) complete disease-specific QOL questionnaires (e.g., PBC-40) would also help substantiate results.

As it takes many years for outcomes to occur in PSC patients, there is a need for surrogate endpoints in clinical trials [2]. This study points towards associations between PROs, risk measures, and disease severity. Further validation would mean that PROs could be used to add value to evaluating new treatments. In addition, understanding symptom burden and the lived experience facilitates the development of diverse approaches to tackling symptoms.

In summary, characterising QOL is key to improving the PSC patient experience and evaluating new therapies. This study provides a strong basis for further research. However, larger longitudinal studies are needed to investigate the association between PROs and disease.