Author information
1
Center For Liver Disease, Department of Medicine, Inova Fairfax Hospital, Falls Church, VA, United States; Betty and Guy Beatty Center for Integrated Research, Inova Health System, Falls Church, VA, United States. Electronic address: zobair.younossi@inova.org.
2
Center for Outcomes Research in Liver Diseases, Washington, DC, USA.
3
Center For Liver Disease, Department of Medicine, Inova Fairfax Hospital, Falls Church, VA, United States; Center for Outcomes Research in Liver Diseases, Washington, DC, USA.
Abstract
BACKGROUND & AIMS:
The chronic liver disease questionnaire for non-alcoholic steatohepatitis (CLDQ-NASH) was developed in a systematic manner for assessment of patient-reported outcomes. This instrument collects data on 36 items grouped into 6 domains: abdominal symptoms, activity/energy, emotional health, fatigue, systemic symptoms, and worry. We aimed to validate CLDQ-NASHin a large group of patients with NASH.
METHODS:
We collected data from patients with biopsy-proven NASH enrolled in 2 international phase 3 trials of selonsertib (NCT03053050 and NCT03053063). Our final analysis comprised 1667 patients who completed the CLDQ-NASH (58±9 years old, 40% male, 52% with cirrhosis, and 69% type 2 diabetes). The CLDQ-NASH was administered before treatment initiation. A standard PRO instrument validation pipeline with internal consistency and validity assessment was applied.
RESULTS:
The domains of CLDQ-NASH demonstrated good to excellent internal consistency: Cronbach's alpha values were 0.80-0.94 and item-to-own domain correlations above 0.50 for 33/36 items. All items correlated to the greatest extent with their own domains (discriminant validity). Known groups validity tests indicated that the instrument consistently discriminated between patients with NASH based on the presence of cirrhosis (vs bridging fibrosis; all but 1 P value <.02), obesity (all but 1 P value<.001), psychiatric comorbidities (all P values<.0001), fatigue (all P values<.001), type 2 diabetes (all but 1 P value<.01). Of the CLDQ-NASH domains, the highest correlated domains with the SF-36 were as follows: physical functioning for activity (rho=.70), mental health for emotional (rho=.72), vitality for fatigue (rho=.75), and body pain for systemic (rho=.72) (all P values<.0001). In contrast, the domains of abdominal and worry, which are disease-specific, did not correlate with the domains in SF-36 (all rho≤.50).
CONCLUSION:
We validated the CLDQ-NASH an analysis of data from 1667 patients with biopsy-proven NASH enrolled in phase 3 trials, observing excellent psychometric characteristics of the instrument.