Characteristics and Outcomes Reported by Patients With Primary Sclerosing Cholangitis Through an Online Registry

BACKGROUND & AIMS: Primary sclerosing cholangitis (PSC) is a fibrostenosing disease of the bile ducts associated with inflammatory bowel disease (IBD), for which the only treatment is liver transplantation. PSC progression has been defined in cohorts from referral centers or single-nation population databases. However, observations made from these cohorts have limited applicability owing to referral bias and demographic confounders. We analyzed data from the worldwide PSC Partners Patient Registry, an international online database established in 2014 to obtain information from individuals with PSC or their caretakers and compare symptoms, disease progression, and treatments of PSC in the United States and other countries. METHODS: We analyzed demographic and clinical characteristics, symptoms, and clinical outcomes of patients with PSC using the PSC Partners Patient Registry. Participants completed an online standardized questionnaire and electronic case report, providing information on age, age at symptom onset, age at PSC diagnosis, methods of diagnosis, concurrent diagnoses, family history, and medication use. RESULTS: Of 873 registrants, 811 (92.9%) had completed questionnaires and 528 (65.1%) had their PSC diagnosis confirmed; we found no significant demographic or clinical differences between patients with vs without a confirmed diagnosis. In contrast to other studies, we found a higher proportion of individuals with PSC to be female (52.5%). However, the mean age at PSC diagnosis (32.4 +/- 14.7 y) and the proportion of individuals with PSC and IBD (67.1%) were similar to those from prior reports. Most cases in the database were from the United States (74.9%). More than half of the participants reported having pruritus, abdominal pain, fatigue, or sleep disturbances; rates were not significantly different among participants within vs outside the United States. There was no significant difference in treatment with ursodeoxycholic acid between participants within vs outside the United States (50.0% and 57.8%; P = .07). The median time of transplant-free survival was 21 years; transplant-free survival was associated with female sex and Crohn's disease. CONCLUSIONS: Our findings from an analysis of data from the PSC Partners Patient Registry confirm those from previous studies, although we found a higher proportion of individuals with PSC to be female. In addition to allowing efficient collection of patient-reported outcomes, the patient-driven registry allows for inclusion of previously under-represented cases of PSC.