Primary Sclerosing Cholangitis (PSC)

Welcome to the online home of the Chris M. Carlos and Catherine Nicole Jockisch Carlos Endowment for Primary Sclerosing Cholangitis (PSC). Thanks to support provided by this endowment, dedicated Mayo Clinic investigators and their teams are making strides to better understand and treat PSC patients, with the ultimate goal of developing a cure for this disease.

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Mon, Jun 3 9:53am

PSC literature elsewhere: Ursodeoxycholic acid therapy in pediatric PSC

By Konstantinos N. Lazaridis, M.D., @klazaridis

2019-05-31 Patient taking medicine

Recently, we have made the decision to include periodically in our PSC blog commentaries of selected clinical studies published by other groups. This tactic aims at keeping the followers of this blog informed about current efforts of the academic community at large to better understand and treat PSC.

In a retrospective study published in the Journal of Pediatrics in June 2019 (see below), Mark Deneau et al., examined the clinical factors predicting normalization of gamma glutamyltransferase (GGT) in children affected by PSC following therapy with ursodeoxycholic acid (UDCA).

In this large study, 344 PSC patients from 46 medical centers were identified with a serum GGT more than 50IU/L at the time of diagnosis who had also repeat testing one year later. Subsequently, 81 patients were excluded because they were not taking UDCA or developed complications of the disease 3 months following the diagnosis. The remaining 263 patients (60% were male with a median age of 12.1 years) were followed for a median of 5.4 years. The median dose of UDCA was 15mg/kg daily. 122 of 263 patients (46%) had normalization of GGT at 1 year. At the time of diagnosis, the group of patients that had favorable biochemical response on UDCA had a lower prevalence of Crohn’s disease, lower total bilirubin, lower aspartate aminotransferase to platelet ratio index, and greater serum albumin level. The presence of small or large duct PSC, or the concurrence of autoimmune hepatitis was not associated with normalization of GGT. The 5-year survival of patients with normalization of UDCA while on UDCA was 99% compared to 77% of patients who did not achieve normalization.

This was a large study from a diverse number of medical centers and the authors should be congratulated for this significant effort. It appears that UDCA normalizes GGT in a number of pediatric patients and these have identifiable clinically relevant factors. The study also suggests that lack of GGT normalization, while on UDCA, should make providers to discontinue therapy although compliance with this drug was unknown. The limitations of the study include its retrospective nature, the fact that it did not account for severity and duration of disease, as well as concurrent immunosuppressive therapy of the patients. In summary, this is an interesting study but we still need better objective biomarkers of PSC severity in order to improve our knowledge on treatment response in PSC patients.

The pdf of the published article can be found here.

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