Surrogate endpoints for clinical trials in primary sclerosing cholangitis: Review and results from an International PSC Study Group consensus process

Cyriel Y. Ponsioen, Roger W. Chapman, Olivier Chazouillères, Gideon M. Hirschfield, Tom H. Karlsen, Ansgar W. Lohse, Massimo Pinzani, Erik Schrumpf, Michael Trauner, Gregory J. Gores – 29 September 2015 – Primary sclerosing cholangitis (PSC) is a rare, but serious, cholestatic disease for which, to date, no effective therapy exists to halt disease progression toward end‐stage liver disease. Clinical trial design to study drugs that improve prognosis is hampered by the relatively low event rate of clinically relevant endpoints.

Engineered fibroblast growth factor 19 reduces liver injury and resolves sclerosing cholangitis in Mdr2‐deficient mice

Mei Zhou, R. Marc Learned, Stephen J. Rossi, Alex M. DePaoli, Hui Tian, Lei Ling – 29 September 2015 – Defects in multidrug resistance 3 gene (MDR3), which encodes the canalicular phospholipid flippase, cause a wide spectrum of cholangiopathy phenotypes in humans. Mice deficient in Mdr2 (murine ortholog of MDR3) develop liver diseases that closely reproduce the biochemical, histological, and clinical features of human cholangiopathies such as progressive familial intrahepatic cholestasis and primary sclerosing cholangitis.

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