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Medical Management of Polycystic Liver Disease: A Position Statement From the European Reference Network on Hepatological Diseases

ERN RARE-LIVER · 2025 · Europe

Source and licence

Authors
Sophia Heinrich, Andreia Margarida Carvalho de Matos, Jordi Colmenero et al.
Title
Medical Management of Polycystic Liver Disease: A Position Statement From the European Reference Network on Hepatological Diseases
Journal
Liver International, 2025
DOI
10.1111/liv.70451
Licence
CC BY 4.0
Changes
Verbatim excerpts; formatting adapted

Full text retrieved 2026-10-27 from Europe PMC · PMC12670357

Licence statement of the original publication

This is an open access article under the terms of the http://creativecommons.org/licenses/by/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.

This page reproduces excerpts of the original publication; the original is authoritative. It has not been reviewed by a physician, does not state a recommendation of Medelix and does not replace medical advice. The guideline may have been updated since.

Open the original publication →Full text on Europe PMC

Recommendations

Verbatim, with grade of recommendation or strength of consensus where given.

Recommendations

  • 1Patients with Liver volume‐related symptoms in non‐localised disease should be considered for SSA treatment. The assessment of symptoms should be done by disease specific questions, TLV measurement and expected TLV trajectoryLevel of recommendation: Strong
  • 2SSA therapy can be considered in symptomatic patients regardless of renal function, although degree of experience from RCTs is limited in advanced CKD (eGFR < 30 mL/min)Level of recommendation: Weak
  • 2SSA can be offered to symptomatic patients with history of cyst infection and/or cholelithiasis with careful subsequent monitoringLevel of recommendation: Weak
  • 3Symptomatic burden should be assessed by validated disease specific PROMS (e.g., PLD‐Q or POLCA), TLV (CT/MRI with volumetric analysis) and if available, expected TLV trajectory before treatment startLevel of recommendation: Strong
  • 4Typical treatment thresholds could include POLCA ≥ 14, PLD‐Q ≥ 32, progression group ≥ 2, or hTLV > 1600 mL/m (KIM ≥ 2)Level of recommendation: Weak
  • 5Treatment should be started at full dose according to clinical trials (Lanreotide 120 mg, Octreotide 40 mg). Dose should be adjusted according to side effects. Patients should be informed about possible side effectsLevel of recommendation: Strong
  • 6The ultimate goal of SSA treatment in PLD is the relief of symptoms related to liver volumeLevel of recommendation: Strong
  • 7PROMS and TLV measurements can be continued at follow‐up to document further stabilisation of the diseaseLevel of recommendation: Strong
  • 8Symptom relief without hTLV progression is indicative of treatment response within the first 6–12 months (e.g., PROM at least after 6 months, hTLV measurements at least after 12 months)Level of recommendation: Strong
  • 9SSA may be continued (as long as benefits overweight side effects) until an attenuation of disease progression by natural disease course is expectedLevel of recommendation: Strong
  • 10Stop when the treatment response criteria are no longer being achievedLevel of recommendation: Strong
  • 11Patients should be aware of side effects before starting treatment and informed that gastrointestinal abnormalities, especially steatorrhea, are common side effects, that can be ameliorated by avoiding fatty food and will disappear spontaneously later onLevel of recommendation: Weak
  • Which patients are eligible for SSA treatment?
  • Are there contraindications to SSA treatment in polycystic liver disease?
  • Which (clinical) parameters are needed to inform the decision to start somatostatin analogue treatment?
  • What are the thresholds of the parameters to start treatment?
  • What are the goals (outcome measures/endpoints) of SSA treatment for polycystic liver disease?
  • What are the parameters to assess response on follow‐up
  • How long do we need to treat patients with SSA?
  • What are the thresholds of the parameters to stop treatment?
  • Which adverse events are common and how do we manage them?

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.

Contents of the original publication

The section headings of the original.

  • Introduction
  • Methods
  • Results
  • Recommendations
  • Future Research Agenda
  • Discussion
  • Supporting information

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