Polycystic Liver Disease Registry (UK)

Recruiting

• Conditions: Polycystic Liver Disease

• Registration Number: NCT04645251
• Lead Sponsor: Portsmouth Hospitals NHS Trust

Brief Summary

Multicentre, observational registry studying the natural course of polycystic liver disease.

Detailed Description

The Polycystic Liver Disease (PLD) registry (UK) is the UK specific part of an international, multicentre, observational registry. The overall international PLD registry is led by Radboud University Medical Center, Nijmegan, Netherlands. Note- please direct any queries about the international registry to the team at Radboud University Medical Center.

All patients with Polycystic Kidney Disease (PKD) with PLD or patients with Isolated (without PKD) Polycystic Liver Disease are eligible for inclusion (\>10 liver cysts).

Data will be collected prospectively and retrospectively including a specific validated PLD questionnaire (PLD-Q).

This registry provides us insight in patient characteristics, risk factors, symptoms, quality of life and treatment strategies in the biggest international PLD cohort so far. Results of this registry will be published and shared at national and international congresses.

Study Timeline

• Study First Submitted: 2020-11-11
• Study First Submitted QC: 2020-11-19
• Study First Posted: 2020-11-27
• Study Start: 2021-01-21
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-12
• Last Update Posted: 2024-11-14
• Primary Completion: 2030-12-01
• Study Completion: 2030-12-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 500

Inclusion Criteria

• Patients with PLD with more than 10 liver cysts
• Patients with PKD and PLD with more than 10 liver cysts

Exclusion Criteria

• Patients with PKD or PLD with less than 10 liver cysts

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Map the natural course of PLD	10 years	To establish the demographics, presence and severity of symptoms (quantified using the validated PLD-Q questionnaire), liver cyst characteristics on imaging (assessed using Qian's classification), biochemical marker abnormalities (including gamma-glutamyl transferase and alkaline phosphatase), rate and nature of complications, need for treatment, change in liver volume in patients with PLD. This data will be collected retrospectively and prospectively from clinical records and the PLD-Q questionnaire.

Secondary Outcome Measures

Name	Time	Method
Determine whether rate of liver growth can be used to predict disease progression	10 years	Liver growth rate will be determined using change in liver volume on serial radiology imaging. This will be compared against patients symptoms (measured via PLD-Q questionnaire), development of complications and need for treatment to establish whether there is a correlation in rate of liver growth and disease progression
Assess quality of life using the validated PLD questionnaire (PLD-Q)	10 years	The PLD-Q questionnaire consists of 18 items assessing symptom frequency and severity. It gives a score (0-100) based on the patient's answers with a higher score indicating more severe, symptomatic disease with a poorer quality of life.
Elucidate risk factors for developing a complication in PLD	10 years	For patients that develop a complication of PLD (including jaundice, abdominal hernia, features of portal hypertension, cyst haemorrhage and cyst infection) we will look through their clinical record to establish if there any risk factors which are common to those with a complication (e.g. age, gender, medications, cyst phenotype).
Elucidate elements that affect PLD progression	10 years	For patients that have a worsening of their PLD (based on increasing symptoms as per the PLD-Q questionnaire, development of complications or need for treatment) we will look through their clinical records to establish if there any elements which are common to those with progressive disease (e.g. age, gender, medications).
Compare effectiveness of different therapies	10 years	For patients who have had or require treatment for PLD we will compare the effectiveness of that treatment based on change in symptoms (based on PLD-Q questionnaire), recurrence of symptoms, development of complications or need for further treatment.

Trial Locations

Locations (2)

Portmouth Hospitals University NHS Trust; 🇬🇧 Portsmouth, Hampshire, United Kingdom

Radboud University Medical Center; 🇳🇱 Nijmegen, Netherlands