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Development and validation of a primary sclerosing cholangitis-specific patient-reported outcomes instrument: The PSC PRO.
Younossi, Zobair M; Afendy, Arian; Stepanova, Maria; Racila, Andrei; Nader, Fatema; Gomel, Rachel; Safer, Ricky; Lenderking, William R; Skalicky, Anne; Kleinman, Leah; Myers, Robert P; Subramanian, G Mani; McHutchison, John G; Levy, Cynthia; Bowlus, Christopher L; Kowdley, Kris; Muir, Andrew J.
Afiliação
  • Younossi ZM; Betty and Guy Beatty Center for Integrated Research, Inova Health System, Falls Church, VA.
  • Afendy A; Department of Medicine, Center for Liver Diseases, Inova Fairfax Hospital, Falls Church, VA.
  • Stepanova M; Center for Outcomes Research in Liver Diseases, Washington, DC.
  • Racila A; Center for Outcomes Research in Liver Diseases, Washington, DC.
  • Nader F; Center for Outcomes Research in Liver Diseases, Washington, DC.
  • Gomel R; Center for Outcomes Research in Liver Diseases, Washington, DC.
  • Safer R; PSC Partners Seeking a Cure, Greenwood Village, CO.
  • Lenderking WR; PSC Partners Seeking a Cure, Greenwood Village, CO.
  • Skalicky A; Evidera, Outcomes Research Division, Bethesda, MD.
  • Kleinman L; Evidera, Outcomes Research Division, Bethesda, MD.
  • Myers RP; Evidera, Outcomes Research Division, Bethesda, MD.
  • Subramanian GM; Gilead Sciences, Inc., Foster City, CA.
  • McHutchison JG; Gilead Sciences, Inc., Foster City, CA.
  • Levy C; Gilead Sciences, Inc., Foster City, CA.
  • Bowlus CL; University of Miami School of Medicine, Miami, FL.
  • Kowdley K; University of California Davis School of Medicine, Sacramento, CA.
  • Muir AJ; Swedish Medical Center, Seattle, WA.
Hepatology ; 68(1): 155-165, 2018 07.
Article em En | MEDLINE | ID: mdl-29152767
Primary 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). CONCLUSION: PSC PRO is a self-administered disease-specific instrument developed according to U.S. Food and Drug Administration guidelines. This preliminary validation study suggests good psychometric properties. Further validation of the instrument in a larger and more diverse sample of PSC patients is needed. (Hepatology 2018;68:155-165).
Assuntos

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Colangite Esclerosante / Medidas de Resultados Relatados pelo Paciente Tipo de estudo: Guideline / Qualitative_research Limite: Adult / Female / Humans / Male / Middle aged Idioma: En Revista: Hepatology Ano de publicação: 2018 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Colangite Esclerosante / Medidas de Resultados Relatados pelo Paciente Tipo de estudo: Guideline / Qualitative_research Limite: Adult / Female / Humans / Male / Middle aged Idioma: En Revista: Hepatology Ano de publicação: 2018 Tipo de documento: Article