Thanks to your active participation in the Registry, significant progress has been made in PSC research. Four publications using Registry data were released in 2024 - two journal articles and two scientific posters. Topics include improving patient-centered clinical trial development, special considerations of pediatric PSC patients, and better understanding PSC-related fatigue and cholangitis.
Click on each title to access the abstract and full article/poster (if available).
Using the Registry’s 2021-2023 “Our Voices” survey results, researchers looked at patient priorities in drug development and factors/barriers contributing to clinical trial participation. While 61% of patients with PSC reported interest in trial participation, only 26% have ever been asked. Conversations and education with/from hepatologists and gastroenterologists “are vital to closing the gap between trial interest and participation”.
Also drawn from the “Our Voices” survey, this study summarizes responses from 51 pediatric PSC patients and caregivers. The responses reveal “a substantial patient/caregiver-reported symptom burden for children with PSC that impacts quality of life and limits access to clinical trials.”
A series of in-depth interviews were conducted via recruitment from Registry participants. Analysis of presence, frequency, severity, and distress related to PSC symptoms led to fatigue emerging as a “prominent and distressing symptom.” These interviews inform development of a symptom assessment tool (survey) for use in clinical trials to measure the effectiveness of a treatment in improving patient-reported outcomes, including fatigue.
Using the “PSC Flare or Cholangitis Attack Patient Survey”, the Registry Team worked directly with an international team of researchers to characterize patient experiences with cholangitis attacks (acute cholangitis). This survey demonstrated a “broad dynamic range of the frequency and severity of symptoms”, and is a first step in developing a precise definition of acute cholangitis for use in clinical trials as a potential outcome of interest.
Many thanks again to the Registry participants who have taken the surveys to make these publications possible! The “PSC Flare or Cholangitis Attack Patient Survey” is currently active in the Registry. Log in and complete this survey today! Data is always de-identified and your responses remain confidential while advancing PSC research.
