Clinical Features of Portal Hypertension in Patients with Rare and Autoimmune Liver Diseases
Rare and autoimmune liver diseases (RALIDs) form a unique and heterogenous group of different disease entities which are usually shadowed by highly prevalent etiologies like viral hepatitis, alcoholic and non-alcoholic liver disease. Affected patients often rely on the expertise of third level medical care facilities for proper diagnosis and treatment and may equally progress to different stages of advanced chronic liver disease (ACLD) and clinically significant portal hypertension (CSPH). CSPH summarizes different clinical criteria associated with increased portal pressure (presence of esophageal varices and other portosystemic collaterals, splenomegaly, ascites, hepatic encephalopathy and an increased hepatic venous pressure gradient (≥ 10mmHg)) and serves as an important predictor for future hepatic decompensation and transplant-free survival. Non-invasive and easily applicable risk scores are equally important for quick prognostic estimations during clinical routine, however, most of these were only validated in highly prevalent disease entities, so far.
The main aim of this thesis is to investigate the prognostic impact of different ACLD stages and distinct features of portal hypertension in RALIDs, which have not been adequately covered so far. For this purpose, three different retrospective analyses will be performed on three different rare disease entities (Primary biliary cholangitis (PBC), Wilsons’ disease (WD) and Autoimmune hepatitis (AIH)), each representing a different pathophysiologic pathway (cholestatic, metabolic and autoimmunologic).
Investigating distinct features of CSPH as well as the prognostic impact of different ACLD stages, non-invasive as well as easily applicable prognostic scores in RALIDs, will improve decision making during clinical routine regarding further diagnostic and therapeutic interventions (e.g. Screening Gastroscopy, Liver vein catheter (LVC), prescription of NSBBs, TIPS, transplantation) and allow for evidence based prognostic statements.
Methods and Skills:
Clinical studies; biomedical statistics; rare liver diseases
Schwaiger E, Burghart L, Signorini L, Ristl R, Kopecky C, Tura A, Pacini G, Wrba T, Antlanger M, Schmaldienst S, Werzowa J, Säemann MD, Hecking M. Empagliflozin in posttransplantation diabetes mellitus: A prospective, interventional pilot study on glucose metabolism, fluid volume, and patient safety. Am J Transplant 19: 907–919, 2019