OBJECTIVES: Nonalcoholic steatohepatitis (NASH) is the most advanced form of nonalcoholic fatty liver disease, characterized by excessive liver fat accumulation, inflammation, cell injury and fibrosis. Here we report on the initial development of the NASH-CHECK; a new NASH-specific patient-reported outcome (PRO) measure.
METHODS: Content for the NASH-CHECK was generated via thematic analyses of semi-structured, concept elicitation (CE) interviews conducted with NASH patients in Virginia, USA. Item selection was guided by a task-force group including clinical experts, patient representatives and PRO researchers. The content validity of the draft measure was assessed through cognitive debriefing (CD) interviews.
RESULTS: Twenty-three CE interviews were conducted (females=18[78%]; age: mean[SD] age=55.9[10.0];range 31.0-73.0 years). Sixteen patients (69.6%) had biopsy-diagnosed NASH (fibrosis grade:F1=1[4.3%];F2=5[21.7%];F3=10[43.5%]); 7 patients (30.4%) had phenotypic-diagnosed NASH. Mean(SD) years since diagnosis=3.9(2.9). Key symptoms reported included pain in upper right abdomen (n=14; 60.9%), fatigue (n=18; 78.3%), poor sleep quality (n=12; 52.2%), cognition problems (impaired memory [n=13; 56.5%]; reduced focus [n=11; 47.8%]) and pruritus [n=10; 43.5%]. Key health-related-quality-of-life (HRQOL) impact included; impaired physical functioning, relationships, emotions (low mood and anxiety), stigma and self-consciousness. The first draft NASH-CHECK included 52 items (16 symptoms/ 36 HRQOL); duplicate items were included to allow patient-selection of most appropriate item phrasing. Fifteen CD interviews were conducted (females=7[46.7%]; age: mean[SD]= 53.6[8.9];range 31.0-68.0 years). Eleven patients (73.3%) had biopsy-diagnosed NASH (fibrosis grade: F1=3[20.0%];F2=2[13.3%];F3=6[40.0%]); 4 patients (26.7%) had phenotypic-diagnosed NASH. Mean (SD) years since diagnosis=3.2(2.8). The NASH-CHECK was reduced to 31-items based on patient preferences for item relevance, acceptability and comprehension. Minor changes were made to the instructions and item wording. The final version was considered relevant and acceptable to the CD interview patients.
CONCLUSIONS: The study was successful in producing a US-English NASH-specific PRO measure assessing symptoms and HRQOL suitable for further psychometric evaluation.
