Patient-reported outcome measures used in patients with primary sclerosing cholangitis: a systematic review

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Abstract

Background

Primary Sclerosing Cholangitis (PSC) is a rare chronic, cholestatic liver condition in which patients can experience a range of debilitating symptoms. Patient reported outcome measures (PROMs) could provide a valuable insight into the impact of PSC on patient quality of life and symptoms. A previous review has been conducted on the quality of life instruments used in liver transplant recipients. However, there has been no comprehensive review evaluating PROM use or measurement properties in PSC patients’ to-date. The aim of the systematic review was to: (a) To identify and categorise which PROMs are currently being used in research involving the PSC population (b) To investigate the measurement properties of PROMs used in PSC.

Methods

A systematic review of Medline, EMBASE and CINAHL, from inception to February 2018, was undertaken. The methodological quality of included studies was assessed using the Consensus-based Standards for selection of health Measurement Instruments (COSMIN) checklist.

Results

Thirty-seven studies were identified, which included 36 different PROMs. Seven PROMs were generic, 10 disease-specific, 17 symptom-specific measures and 2 measures on dietary intake. The most common PROMs were the Short form-36 (SF-36) ( n = 15) and Chronic liver disease questionnaire (CLDQ) ( n = 6). Only three studies evaluated measurement properties, two studies evaluated the National Institute of Diabetes Digestive and Kidney Diseases Liver Transplant (NIDDK-QA) and one study evaluated the PSC PRO; however, according to the COSMIN guidelines, methodological quality was poor for the NIDDK-QA studies and fair for the PSC PRO study.

Conclusion

A wide variety of PROMs have been used to assess health-related quality of life and symptom burden in patients with PSC; however only two measures (NIDDK-QA and PSC PRO) have been formally validated in this population. The newly developed PSC PRO requires further validation in PSC patients with diverse demographics, comorbidities and at different stages of disease; however this is a promising new measure with which to assess the impact of PSC on patient quality of life and symptoms.

Electronic supplementary material

The online version of this article (10.1186/s12955-018-0951-6) contains supplementary material, which is available to authorized users.

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Most cited references 43

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PSC-IBD: a unique form of inflammatory bowel disease associated with primary sclerosing cholangitis.

William J Sandborn, Alan R Zinsmeister, Edward V Loftus … (2004)

Inflammatory bowel disease associated with primary sclerosing cholangitis (PSC-IBD) may have a high prevalence of rectal sparing, backwash ileitis, and colorectal neoplasia. To describe the clinical features and outcomes of PSC-IBD and compare these to a group of chronic ulcerative colitis (CUC) patients. The medical records of all patients with PSC-IBD evaluated at the Mayo Clinic Rochester between 1987 and 1992 were abstracted for information on endoscopic and histological features, colorectal neoplasia, surgery, and other clinical outcomes. Patients referred for colorectal neoplasia and those who did not undergo colonoscopy with biopsies were excluded. A control group of CUC patients matched for sex, duration of IBD at first clinic visit, and calendar year of first clinic visit was identified, and similar information was abstracted. Seventy one PSC-IBD patients and 142 CUC patients without PSC were identified. Rectal sparing and backwash ileitis were more common in the PSC-IBD group (52% and 51%, respectively) than in controls (6% and 7%, respectively). Overall, colorectal neoplasia developed in 18 cases and 15 controls, including 11 cancers (seven cases and four controls). An increased risk of colorectal neoplasia or death was not detected in a matched analysis. Although the cumulative incidence of colorectal neoplasia was higher in cases (33%) than in controls (13%) at five years, this was of borderline statistical significance (p=0.054, unmatched log rank test). Overall survival from first clinic visit was significantly worse among cases (79% v 97%) at five years (p<0.001, unmatched log rank test). PSC-IBD is frequently characterised by rectal sparing and backwash ileitis. Colorectal neoplasia develops in a substantial fraction and overall survival is worse. PSC-IBD may represent a distinct IBD phenotype.

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Pouchitis after ileal pouch-anal anastomosis for ulcerative colitis occurs with increased frequency in patients with associated primary sclerosing cholangitis.

C. Penna, R. Dozois, W Tremaine … (1996)

Primary sclerosing cholangitis (PSC), present in 5% of patients with ulcerative colitis, may be associated with pouchitis after ileal pouch-anal anastomosis. The cumulative frequency of pouchitis in patients with and without PSC who underwent ileal pouch-anal anastomosis for ulcerative colitis was determined. A total of 1097 patients who had an ileal pouch-anal anastomosis for ulcerative colitis, 54 with associated PSC, were studied. Pouchitis was defined by clinical criteria in all patients and by clinical, endoscopic, and histological criteria in 83% of PSC patients and 85% of their matched controls. PSC was defined by clinical, radiological, and pathological findings. One or more episodes of pouchitis occurred in 32% of patients without PSC and 63% of patients with PSC. The cumulative risk of pouchitis at one, two, five, and 10 years after ileal pouch-anal anastomosis was 15.5%, 22.5%, 36%, and 45.5% for the patients without PSC and 22%, 43%, 61%, and 79% for the patients with PSC. In the PSC group, the risk of pouchitis was not related to the severity of liver disease. In conclusion, the strong correlation between PSC and pouchitis suggest a common link in their pathogenesis.

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Health-related quality of life in chronic liver disease: the impact of type and severity of disease.

Zobair Younossi, M Kiwi, L. Price … (2001)

The type and severity of chronic liver disease may have different effects on health-related quality of life (HRQL). The aim of our study was to determine whether HRQL in patients with chronic liver disease differs by type and severity of disease and to identify which clinical and physiological factors affect this impairment. In this study, HRQL was measured with a generic (Short Form 36) and a liver disease-specific (Chronic Liver Disease Questionnaire) questionnaire. Clinical, demographic, and laboratory data were collected at office visits. Patient's HRQL scores were compared with the published norms and to the chronically ill populations. A total of 353 patients (mean age 50 yr, 51% men) with chronic liver disease, either viral disease (hepatitis B and C), cholestatic disease (primary biliary cirrhosis or primary sclerosing cholangitis), or hepatocellular disease were enrolled in the study. In general, HRQL in patients with chronic liver disease was lower than the normal population and was similar to that of patients with chronic obstructive pulmonary disease or congestive heart failure. In cirrhotic patients, some dimensions of HRQL were less impaired in patients with cholestatic disease than in those with hepatocellular diseases. More severe disease (higher Child's class) was associated with a lower Chronic Liver Disease Questionnaire score and the Short Form 36's physical component summary scores. Older age had a weak negative association with the physical aspects of HRQL. We conclude that chronic liver disease substantially reduces HRQL, and this impact does not differ markedly by type of disease. Older age and measures of disease severity were associated with poorer HRQL.

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Author and article information

Contributors

Fatima Isa: Fatima.isa@PHE.gov.uk

Grace M. Turner: G.Turner.1@bham.ac.uk

Geetinder Kaur: G.Kaur.2@bham.ac.uk

Derek Kyte: D.G.Kyte@bham.ac.uk

Anita Slade: A.L.Slade@bham.ac.uk

Tanya Pankhurst: Tanya.Pankhurst@uhb.nhs.uk

Larissa Kerecuk: Larissa.Kerecuk@bch.nhs.uk

Thomas Keeley: Thomas.Keeley@PAREXEL.com

James Ferguson: James.Ferguson@uhb.nhs.uk

Melanie Calvert: M.Calvert@bham.ac.uk

Journal

Journal ID (nlm-ta): Health Qual Life Outcomes

Journal ID (iso-abbrev): Health Qual Life Outcomes

Title: Health and Quality of Life Outcomes

Publisher: BioMed Central (London )

ISSN (Electronic): 1477-7525

Publication date (Electronic): 5 July 2018

Publication date PMC-release: 5 July 2018

Publication date Collection: 2018

Volume: 16

Electronic Location Identifier: 133

Affiliations

[1 ]Public Health England, 5 St Philips Place, Birmingham, B3 2PW UK

[2 ]ISNI 0000 0004 1936 7486, GRID grid.6572.6, Centre for Patient Reported Outcomes Research (CPROR), Institute of Applied Health Research, , University of Birmingham, Edgbaston, ; Birmingham, B15 2TT UK

[3 ]NIHR Birmingham Biomedical Research Centre, Birmingham, B15 2TT UK

[4 ]ISNI 0000 0004 0376 6589, GRID grid.412563.7, University Hospital Birmingham, ; Birmingham, B15 2TH UK

[5 ]ISNI 0000 0004 0399 7272, GRID grid.415246.0, Birmingham Children’s Hospital, ; Birmingham, B4 6NH UK

[6 ]ISNI 0000 0004 0616 2801, GRID grid.477778.c, PAREXEL International, ; Evergreen House North, 160 Euston Road, London, NW1 2DX UK

Article

Publisher ID: 951

DOI: 10.1186/s12955-018-0951-6

PMC ID: 6034220

PubMed ID: 29976215

SO-VID: 7b179531-b8cc-4d15-8813-ce6097873a5d

License:

Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License ( http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver ( http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated.

History

Date received : 21 September 2017

Date accepted : 4 June 2018

Funding

Funded by: Metchley Park Medical Society

Custom metadata

ScienceOpen disciplines: Health & Social care

Keywords: primary sclerosing cholangitis,cholestasis,patient reported outcome measures (proms),prospero (registration number: crd42016036544).

Data availability:

ScienceOpen disciplines: Health & Social care

Keywords: primary sclerosing cholangitis, cholestasis, patient reported outcome measures (proms), prospero (registration number: crd42016036544).

Patient-reported outcome measures used in patients with primary sclerosing cholangitis: a systematic review

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Most cited references 43

PSC-IBD: a unique form of inflammatory bowel disease associated with primary sclerosing cholangitis.

Pouchitis after ileal pouch-anal anastomosis for ulcerative colitis occurs with increased frequency in patients with associated primary sclerosing cholangitis.

Health-related quality of life in chronic liver disease: the impact of type and severity of disease.

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