Authors: Holly Payton Shifman et al.

Journal: Hepatology. 78(S1):S1-S2154, October 2023. https://journals.lww.com/hep/toc/2023/10001

Abstract:

Background: Primary sclerosing cholangitis (PSC) is a rare, chronic, cholestatic liver disease with no FDA-approved treatments available. Research focusing on pediatric patients with PSC is limited. We sought to characterize symptom burden and barriers to clinical trial enrollment for pediatric PSC patients and their caregivers.

Methods: We analyzed survey responses from pediatric PSC patients and their caregivers using the PSC Partners Patient Registry Our Voices survey. This registry, created in 2014, collects data on patient demographics, disease status, concurrent diagnoses, medications, clinical trial enrollment, and quality-of-life measures with the goal of incorporating patient experiences and priorities into drug development and clinical trial design.

Results: Our analysis included 51 surveys (28M/23F) from pediatric PSC patients and their caregivers (90% caregivers, 10% patients). Most children were between 13 and 17 years old at the time of survey completion (67%), and the majority self-identified as White (88%) and non-Hispanic/non-Latino (88%). The most common symptoms reported by children include: fatigue (71%), abdominal pain (69%), anxiety (59%), loss of appetite (51%), insomnia (49%), and pruritis (45%). When experiencing symptoms at their worst, over half of the patients reported limitations in physically demanding activities (67%), work/school duties (63%), social life activities (55%), and less physically demanding activities for fun or exercise (53%). While more than 50% of patients/caregivers expressed willingness to participate in clinical trials, none of these patients reported current or previous enrollment in trials for new or investigational PSC drugs. Only one patient reported ever being asked to participate in a trial for investigational treatment of PSC.

Conclusion: This study revealed substantial symptom burden among pediatric PSC patients and impact on daily activities and quality of life, as reported by both patients and caregivers. Despite the majority expressing willingness to participate in clinical trials, none had done so, indicating unmet needs in trial availability and accessibility for this population. Future efforts should focus on developing patient-centered clinical endpoints, increasing trial availability for pediatric PSC patients, and reducing logistical barriers to trial involvement.