Medical management of polycystic liver disease: a position statement from the European Reference Network on Hepatological Diseases
Heinrich, Sophia de Matos, Andreia Margarida Carvalho Colmenero, Jordi Elsharkawy, Ahmed M Fraňková, Soňa Halbritter, Jan Mrzljak, Anna Müller, Roman-Ulrich Pereira, Rafaela Strnad, Pavel
Heinrich, Sophia
de Matos, Andreia Margarida Carvalho
Colmenero, Jordi
Elsharkawy, Ahmed M
Fraňková, Soňa
Halbritter, Jan
Mrzljak, Anna
Müller, Roman-Ulrich
Pereira, Rafaela
Strnad, Pavel
Affiliation
Hannover Medical School; European Reference Network on Hepatological Diseases; Centro Hospitalar e Universitário de Coimbra; University of Barcelona; Hospital Clínic Barcelona; University Hospitals Birmingham NHS Foundation Trust; University of Birmingham; Institut klinické a experimentální medicíny; Charité Universitätsmedizin Berlin; University Hospital Center Zagreb; University of Zagreb; University of Cologne; University of Cologne; University Hospital Cologne; Hospital Curry Cabral; University Hospital RWTH Aachen; University Hospitals KU Leuven; Medical University of Warsaw; Amsterdam University Medical Center
Other Contributors
Publication date
2025-12-02
Subject
Collections
Research Projects
Organizational Units
Journal Issue
Abstract
Polycystic liver disease (PLD) is a rare genetic disorder characterised by progressive liver enlargement due to multiple cysts. The main symptoms are liver volume-related. Although randomised controlled trials have shown that somatostatin analogues (SSAs) reduce liver volume as well as symptoms, specific guidance on when and how to use SSAs in clinical practice is still lacking. A panel of 15 hepatologists and nephrologists developed practical guidance on SSA use, based on a systematic literature search, expert surveys, and clinical experience. This consensus was reached during a two-day workshop by the European Reference Network on Hepatological Diseases, using 11 predefined key questions and an iterative Delphi process. PLD patients with liver volume-related symptoms, diffuse disease, and significant hepatomegaly are eligible for SSA therapy, regardless of kidney function. Disease burden should be assessed with validated PROMs as well as liver volumetry before and during treatment. Symptom relief without liver growth is a valid treatment response. Therapy may continue until the natural course of disease slows down (e.g., with the onset of menopause). Trials report benefits of SSA therapy for up to 3-4 years. Early discontinuation may cause a rebound of liver volume. As SSA therapy remains off-label, limited access and reimbursement hamper widespread use in Europe. We present state-of-the-art guidance on the practical use of the only available medical therapy for severe PLD including eligibility, start and stop criteria and identifying research gaps. This consensus-based guidance provides much-needed practical recommendations for the use of somatostatin analogues in managing severe polycystic liver disease. By defining eligibility, treatment goals, and monitoring strategies, it supports more standardised, patient-centered care across Europe.
Citation
Heinrich S, de Matos AMC, Colmenero J, Elsharkawy AM, Fraňková S, Halbritter J, Mrzljak A, Müller RU, Pereira R, Strnad P, Teemer CAJ, Verbeek J, Wronka KM, Nevens F, Taubert R, Drenth JPH; European Reference Network on Hepatological Liver Diseases (ERN RARE‐LIVER). Medical Management of Polycystic Liver Disease: A Position Statement From the European Reference Network on Hepatological Diseases. Liver Int. 2026 Jan;46(1):e70451. doi: 10.1111/liv.70451.
Type
Article