• Zobair M Younossi, Arian Afendy, Maria Stepanova, Andrei Racila, Fatema Nader, Rachel Gomel, Ricky Safer, William R Lenderking, Anne Skalicky, Leah Kleinman, Robert P Myers, G Mani Subramanian, John G McHutchison, Cynthia Levy, Christopher L Bowlus, Kris Kowdley, and Andrew J Muir.
• Betty and Guy Beatty Center for Integrated Research, Inova Health System, Falls Church, VA.
• Hepatology. 2018 Jul 1; 68 (1): 155-165.

AbstractPrimary sclerosing cholangitis (PSC) is a chronic liver disease associated with inflammation and biliary fibrosis that leads to cholangitis, cirrhosis, and impaired quality of life. Our objective was to develop and validate a PSC-specific patient-reported outcome (PRO) instrument. We developed a 42-item PSC PRO instrument that contains two modules (Symptoms and Impact of Symptoms) and conducted an external validation. Reliability and validity were evaluated using clinical data and a battery of other validated instruments. Test-retest reliability was assessed in a subgroup of patients who repeated the PSC PRO after the first administration. One hundred two PSC subjects (44 ± 13 years; 32% male, 74% employed, 39% with cirrhosis, 14% with a history of decompensated cirrhosis, 38% history of depression, and 68% with inflammatory bowel disease [IBD]) completed PSC PRO and other PRO instruments (Short Form 36 V2 [SF-36], Chronic Liver Disease Questionnaire [CLDQ], Primary Biliary Cholangitis - 40 [PBC-40], and five dimensions [5-D Itch]). PSC PRO demonstrated excellent internal consistency (Cronbach alphas, 0.84-0.94) and discriminant validity (41 of 42 items had the highest correlations with their own domains). There were good correlations between PSC PRO domains and relevant domains of SF-36, CLDQ, and PBC-40 (R = 0.69-0.90; all P < 0.0001), but lower (R = 0.31-0.60; P < 0.001) with 5-D Itch. Construct validity showed that PSC PRO can differentiate patients according to the presence and severity of cirrhosis and history of depression (P < 0.05), but not by IBD (P > 0.05). Test-retest reliability was assessed in 53 subjects who repeated PSC PRO within a median (interquartile range) of 37 (27-47) days. There was excellent reliability for most domains with intraclass correlations (0.71-0.88; all P < 0.001).© 2017 by the American Association for the Study of Liver Diseases.

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