BACKGROUND AND AIMS: Nonalcoholic steatohepatitis (NASH) is a progressive form of fatty liver disease characterized by excessive liver fat accumulation, inflammation, cell injury and fibrosis. Aim was to develop a NASH-specific patient-reported outcome (PRO) measure (NASH-CHECK) suitable for clinical practice and clinical trials with NASH patients.
METHOD: A NASH conceptual model was developed based on review of published/grey literature, social media patient narratives and medical / patient expert (US/UK) review. The model guided concept elicitation (CE) interviews with NASH patients recruited via a tertiary care centre (USA). NASH-CHECK content was generated via thematic analysis of CE data and review by clinical experts and patient representatives. Cognitive debriefing (CD) interviews evaluated NASH-CHECK content validity.
RESULTS: Literature/social media review confirmed that NASH profoundly impacts patient functioning and health-related-quality-of-life (HRQOL). 23CE/15CD interviews were conducted. CE sample: 18 females; mean age=55.9(31.0-73.0 yrs); biopsy-diagnosed NASH=11(F1=1;F2=5;F3=10); mean yrs since diagnosis=3.9; mean BMI=36.4(26.1-43.3). CD sample: 7 females; mean age=53.6(31.0-68.0 yrs); biopsy-diagnosed NASH=11[F1=3;F2=2;F3=6]; mean yrs since diagnosis=3.2; mean BMI=36.4(26.1-43.3). No patients had evidence of hepatic decompensation. CE interviews: key symptoms reported included pain in upper right abdomen (n=14), fatigue (n=18), poor sleep quality (n=12), and cognition problems (impaired memory [n=13; reduced focus [n=11]); key HRQOL impacts included impaired physical functioning (reduced capacity to walk short distances), ability to conduct daily living tasks (e.g. household chores/personal care), reduced quality of relationships (e.g. pain/fatigue limited willingness/ability to engage in social activities), low mood, anxiety and self-consciousness. The 1st draft NASH-CHECK included 52 items including some duplication to allow patient-selection of preferred phrasing. CE interviews reduced NASH-CHECK to 31-items based on patient preferences for item relevance, acceptability and comprehension. The final version was considered relevant and acceptable to the CD patients.
CONCLUSION: The study was successful in producing a US English NASH-specific PRO measure assessing symptoms and HRQOL (NASH-CHECK) suitable for psychometric evaluation. Further work is underway to translate and validate this tool in UK and other European territories.
