Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease characterized by inflammation and fibrosis of bile ducts, usually medium and large bile ducts, which may progress to end-stage liver disease and liver failure. Several hallmarks distinguish PSC from other liver diseases: 1) A close association with inflammatory bowel disease (IBD) in 70-80% of patients; 2) The neoplastic risk for either liver (cholangiocarcinoma) or colon (colonic cancer; 3) Clinical variants including the overlap syndrome with autoimmune hepatitis; 4) Predominance for male gender in all age groups, with some features unique to children.
The pathogenesis of PSC is still unknown, so PSC remains an orphan disease for which up to now there has been no effective treatment apart from liver transplantation; however, there is a high risk of recurrence in the new graft after transplantation. A genetic predisposition has been recognized, and the role of gut microbioma in the pathogenesis of the disease is still under consideration in terms of pathogenesis and therapeutic management.
PSC is an emerging disease. Its annual incidence rate ranges between 0.7 and 49 cases per million, and its prevalence rate (approximately around 1 per 10,000 inhabitants in Northern Europe) conforms with the definition of rare disease. There is an increasing prevalence/incidence of the disease around the world, due to the new diagnostic tools for imaging. Considering the IBD population, PSC is present approximately in 7.5-8% of patients with ulcerative colitis or Crohn's disease.
There are two important networks for PSC in the world: European Reference Networks (ERN)-RARE-LIVER and the International PSC Study Group (IPSCSG). The ERN-RARE-LIVER is a Europe-wide network for centres of excellence in the clinical management of rare liver disease, including PSC in adults and children. ERN was started in 2017, which functions as a platform to improve healthcare by initiating registries, foster research efforts and coordinate the development of clinical guidelines in Europe. The IPSCSG was founded in Oslo in 2010 with the aim to coordinate PSC research projects between leading institutions worldwide. More than 20 countries in the world are represented and are involved both in basis and clinical research projects.
Finally, in terms of therapeutics, PSC is an area with greatest advances. Several innovative therapies are currently ongoing in phase 2 and phase 3 clinical trials. Based on new pathogenetic concepts, an important therapeutic target is gut microbiota. As a consequence, numerous studies are devoted to antibiotics or faecal microbiota transplantation. However, combination therapy utilizing agents directed against different targets is the most promising strategy for management of PSC.
Pathogenesis of PSC
Immunogenetics of PSC
IBD in PSC
Treatment of PSC
Neoplastic risk for liver and colon
Sclerosing cholangitis in children
Transplant in PSC
1. Giorgina Mieli-Vergani Paediatric Liver, GI and Nutrition Centre, MowatLabs, King's College London Faculty of Life Sciences & Medicine at King's College Hospital, London, UK.
2. Diego Vergani Institute of Liver Studies, MowatLabs, King's College London Faculty of Life Sciences & Medicine at King's College Hospital, London, UK.
3. Angelo Di Giorgio Paediatric Hepatology, Gastroenterology and Transplantation, ASST Hospital Papa Giovanni XXIII, Bergamo, Italy.
4. Nora Cazzagon Department of Surgery, Oncology and Gastroenterology, University of Padova, Padova, Italy.
5. Martina Gambato Department of Surgery, Oncology and Gastroenterology, University of Padova, Padova, Italy.
6. Annarosa Floreani Studiosa Senior, University of Padova, Scientific Consultant for Hepatology, IRCCS Negrar, Verona, Italy.
7. Pietro Invernizzi Division Gastroenterology and Center for Autoimmune Liver Diseases, European Reference Network (ERN) RARE-LIVER Center, San Gerardo Hospital, University of Milano - Bicocca School of Medicine, Monza, Italy.
8. Palak Trivedi National Institute for Health Research Birmingham Biomedical Research Centre and Centre for Liver Research, University of Birmingham, Birmingham, UK.
9. Tobias J. Weismüller Department of Internal Medicine I, University of Bonn, Bonn, Germany.
10. Andrea Tenca Faculty of Medicine, University of Helsinki, Helsinki, Finland.
11. Annika Bergquist Division of Upper GI disease, Karolinska University Hospital, Karolinska Institutet, Stockholm, Sweden.
The list is arranged in no particular order and being updated.
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