BACKGROUND AND AIMS: NASH-CHECK is the first patient-reported outcome measure (PROM) developed specifically for patients with non-cirrhotic non-alcoholic steatohepatitis (NASH; fibrosis F1-F3) in accordance with the US Food and Drug Administration guidance for PROMs supportive of drug labelling claims. It contains items assessing symptoms and health-related quality of life (HRQOL; activity limitations, emotions and lifestyle items). The current study assessed the symptom burden and HRQOL impacts experienced by patients with compensated cirrhotic NASH and compared them to the concepts covered by NASH-CHECK.
METHOD: A qualitative study with concept elicitation interviews was conducted with compensated cirrhotic NASH patients attending hepatology clinics in the United States and United Kingdom. Thematic analysis identified key symptoms and HRQOL impacts. These were compared with the key concepts encompassed by NASHCHECK. New symptom items identified were discussed with an international group of clinicians to gain consensus on their relevance to compensated cirrhotic NASH.
RESULTS: Thirty-three patients were interviewed, 61% female, mean (SD) age = 64.5 (7.8) years, body mass index = 34.5 (4.1). Comorbidities included type II diabetes 81.8%, obesity 87.9%, and hypertension 39.4%. Saturation was reached during the thematic analysis. Key symptoms identified in NASH-CHECK (pain, bloating, itch, fatigue, sleep, cognitive) were all reported. Key HRQOL concepts identified in NASH-CHECK (activity limitations, emotional, social, relationships, and work impacts) were also frequently reported and found to be comprehensive in relation to patients’ experience of NASH with compensated cirrhosis. Additional symptoms reported included muscle cramps 54.6%, generalised pain 42.4%, shortness of breath 42.4%, and heartburn 39.4%. On clinical review, these additional symptoms identified were deemed to be related to comorbidities or medication side-effects and so not directly related to compensated NASH cirrhosis or appropriate for inclusion in a PROM.
CONCLUSION: NASH patients with compensated cirrhosis experience similar symptoms and HRQOL impacts to patients with earlier stages of disease but may experience them more severely. These data suggest that NASH-CHECK is likely to be applicable in the compensated NASH cirrhosis population. Cognitive debriefing interviews are being conducted to further evaluate NASH-CHECK in cirrhotic patients.
