Health-related quality of life and patient-reported outcome measures in NASH-related cirrhosis

Abstract

Background & aims: Non-alcoholic steatohepatitis (NASH) is known to have a negative impact on patients' health-related quality of life (HRQoL), even before progression to cirrhosis has occurred. The burden of NASH-related cirrhosis from the patient perspective remains poorly understood. Herein, we aimed to identify the burden of disease and HRQoL impairment among patients with NASH-related compensated cirrhosis.

Methods: This targeted literature review sought first to identify the humanistic burden of disease from the perspective of patients with diagnosed NASH-cirrhosis and, secondly, to identify generic or disease-specific patient-reported outcome measures (PROMs) used to assess the impact of NASH-cirrhosis. Searches were conducted in bibliographical databases, grey or unpublished literature, liver disease websites, support group websites and online blogs. A quality assessment of specific PROMs was conducted.

Results: Patients with NASH-cirrhosis are reported to suffer from lower HRQoL than patients with non-cirrhotic NASH and the general population with respect to physical health/functioning, emotional health and worry, and mental health. Thirteen PROMs were identified, of which 4 were liver-disease specific: CLDQ, CLDQ-NAFLD, LDQoL and LDSI. The most commonly used measures do not comply with current industry or regulatory standards for PROMs and/or are not validated for use in a cirrhotic NASH population.

Conclusions: Patients with NASH-cirrhosis have lower HRQoL and poorer physical health than patients with non-cirrhotic NASH. However, the literature lacked detail of the everyday impact on patients' lives. Currently, a number of PROMs are available to measure the impact of the disease in patients with chronic liver conditions. The lack of studies that include qualitative insights in this population mandates further exploration and research.

Lay summary: It is not well understood how having non-alcoholic fatty liver disease (NAFLD)-related cirrhosis affects a person's everyday wellbeing and quality of life. Some research has been done with patients who have early stages of liver disease but not people with cirrhosis. We found that patients with NAFLD-related cirrhosis tended to have poorer health than patients without cirrhosis. But there was not very much information from patients themselves and there were no tools or questionnaires just for this group of patients.

Keywords: CLDQ, chronic liver disease questionnaire; COSMIN, The COnsensus-based Standards for the selection of health Measurement INstruments; Cirrhosis; EMA, European Medicines Agency; FDA, United States Food and Drug Administration; FIS, fatigue impact scale; HRQoL, health-related quality of life; LDQoL, liver disease quality of life questionnaire; LDSI, liver disease symptom index; MS, multiple sclerosis; NAFL, non-alcoholic fatty liver; NAFLD; NAFLD, non-alcoholic fatty liver disease; NASH, non-alcoholic steatohepatitis; Non-alcoholic steatohepatitis; PHAQ, patient-reported outcome measurement information system health assessment questionnaire; PRO, patient-reported outcome; PROM, patient-reported outcome measure; QoL, quality of life; RI, researcher interpretation; SF-36, short form health profile 36; health-related quality of life; liver; patient-reported outcome measures.

Graphical abstract

Fig. 1

PRISMA flow diagram: for objective (a) — humanistic burden of NASH-cirrhosis. 3,015 records were identified from databases. After removal of duplicates, 1,809 records were screened. Eligibility assessment of texts, plus 5 grey literature studies, resulted in 31 studies being included in the humanistic burden of NASH-cirrhosis synthesis.

Fig. 2

PRISMA flow diagram: for Objective (b) — PROMs F4. A second database search identified a further 545 studies in addition to search one (objective (a)). After removal of duplicates, 2,355 records were screened. Eligibility assessment of texts resulted in 20 studies being included in the PROMs synthesis.