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

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

Clin Gastroenterol Hepatol. 2018 Apr 26;:

Authors: Kuo A, Gomel R, Safer R, Lindor KD, Everson GT, Bowlus CL

Abstract
BACKGROUND AND 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 due 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 Unites States (US) 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 of symptom onset, age of PSC diagnosis, methods of diagnosis, concurrent diagnoses, family history, and medication use.
RESULTS: Of 873 registrants, 811 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, mean age of PSC diagnosis (32.4 + 14.7 years) and proportion of individuals with PSC and IBD (67.1%) were similar to those from prior reports. Most cases in the database were from the US (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 US. There was no significant difference in treatment with ursodeoxycholic acid between participants within vs outside the US (50.0% and 57.8%, P = 0.07). Median time of transplant-free survival was 21 years; transplant-free survival 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.

PMID: 29705262 [PubMed – as supplied by publisher]

PubMed Link: https://www.ncbi.nlm.nih.gov/pubmed/29705262?dopt=Abstract