2017. Development and validation of a primary sclerosing cholangitis–specific patient‐reported outcomes instrument: The PSC PRO
Plain-Language Summary
There are currently no PSC-specific tools to evaluate patient reported outcomes (PROs). The study team set out to develop and test a 42 item instrument that would capture PSC-specific patient reported symptoms and symptom impact. Recruitment happened at 4 different sites in the US, including through the PSC Partners Patient Registry. This instrument could be used by researchers and clinicians to inform future treatment regimens and aid in monitoring of clinical trials. It can be self-administered and would take approximately 7-15 minutes to complete. Although the development and initial validation was successful, a larger study will be needed to validate its use in a more diverse population.
Authors: 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 Andrew J. Muir
Journal: AASLD Hepatology First published:20 November 2017 https://doi.org/10.1002/hep.29664 https://doi.org/10.1002/hep.29664
Excerpts:
"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."
"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."