Quality of Life

Studying the quality of life (QoL) of patients suffering from rare liver diseases is an important activity of the network.

Quality of Life workshop
on 10 September 2022

The third Quality of Life workshop of ERN-RARE LIVER took place on Saturday, 10 September 2022 in the Radisson Blue Airport Hotel in Hamburg, right after the ERN RARE LIVER members’ meeting. We had 8 presentations on HrQol in several rare liver diseases, including autoimmune hepatitis, vascular liver disease and biliary malformations. Most presentations were focused on quantifying HrQol in rare liver disease, but we also discussed how to identify the most important topics that affect HrQol (unmet clinical needs project in patients) or how to improve HrQol in these patients (social support project). Large steps are taken in the autoimmune liver disease, the main challenge will be how to expand this to the other rare liver diseases and the role of the Qol core group in this process. There were around 25 participants with active discussions. In case you want to see the presentations, please find the link to the protected website here: 

  1. Go to https://webgate.ec.europa.eu/ern/
  2. Login with your EU login (the same one that you use for CPMS), or create a new account 
  3. Access the network RARE-LIVER or proceed with “Search & join networks” 
  4. Click on “Library” – “Meetings” – “Members’ Meeting September 2022” – “QoL Workshop”
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HRQoL PROM repository

The ERN RARE-LIVER Quality of Life (QoL) working group has created a repository of patient-reported outcome measures (PROMs), that are used in studies conducted by the QoL-group. The repository contains an overview of the PROMs as well as available translations. Where PROMs are available from external sources, the links are given. If they were translated within an ERN-project, the translations are provided for download. Please note, that we make no warranties for the accuracy of the documents – use of the provided translations is at own responsibility. If you use a PROM within a project of the ERN RARE-LIVER that is not yet listed in the repository, please feel free to share it with us. 

  1. Open repository: https://webgate.ec.europa.eu/ern/
  2. Login with your EU login (the same one that you use for CPMS), or create a new account 
  3. Access the network RARE-LIVER or proceed with “Search & join networks” 
  4. Click on “Library” – PROM Database

Overview of Studies on HRQoL (as of August 2022)

Several studies are currently underway in the area of quality of life (QoL) in rare liver diseases:

Health related Quality of Life and medication adherence in adult patients with autoimmune hepatitis: a multicentre, cross-sectional study

Background/aims: Characterization of the relationship between HRQL and factors associated with HRQL including anxiety/depression, medication adherence, and clinical features in autoimmune hepatitis (AIH) patients.

Health related Quality of Life and medication adherence in adult patients with autoimmune hepatitis, primary biliary cholangitis, primary sclerosing cholangitis and variant syndromes: a multicentre study

Background/aims: Compare HRQoL of patients with PBC, PSC, Variant Syndromes and AIH. Identify disease specific factors that determine HRQoL.

Q.RARE.LI - Improving health-related quality of life in patients with rare autoimmune liver diseases by structured peer-delivered support: a transnational effectiveness-implementation hybrid trial

Background/aims: The proposed project addresses the unmet psychosocial support needs of patients. We will assess the effectiveness of a peer-delivered psychosocial support intervention (that was developed in Hamburg, Germany by Bernd Löwe and Natalie Uhlenbusch) in routine care and prepare

Health Related Quality of Life (HRQoL) and adherence in adult patients transplanted for Autoimmune Hepatitis (AIH): a pilot study.

Background/aims: A proportion of patients with AIH will require orthotopic liver transplantation (OLTx). The major indication, like in many other chronic liver conditions leading to cirrhosis is decompensation of liver function with features of encephalopathy, variceal bleed or treatment resistant

Health Related Quality of Life (HRQoL) and adherence after during transition from paediatric to adults healthcare in patients with Autoimmune Hepatitis (AIH): a single center study.

Background/aims: A proportion of patients with AIH are diagnosed at a childhood and required transition from paediatric to adult healthcare system. It is known, that the transfer of care from paediatric to adults is a risk factor of non-compliance, thus

Validation of polycystic liver disease questionnaire with 1 week recall period and determination of treatment threshold for polycystic liver disease questionnaire

Background/aims: The polycystic liver disease questionnaire was developed and validated to quantify the symptom severity in patients with polycystic liver disease. Though this questionnaire is currently used in clinical practice and research, the questionnaire does not have a clinical threshold.

Mood disorders in polycystic liver disease

Background/aims: Previous studies demonstrated that polycystic liver disease (PLD) affects quality of life and specifically the mental component. This study aims to further investigate this by determining whether PLD is associated with anxiety or depression and if PLD disease severity

Unmet Clinical Needs (UCN)

Background/aims: This project aims to understand patients’ difficulties in order to quantify and prioritize patients’ problems and needs related to their disease that should be addressed by clinicians and medicine/science/research. The objective is to identify problems as well as potential

Health related Quality of Life in patients with inherited unconjugated hyperbilirubinemia (Crigler-Najjar syndrome): a multicentre study.

Background/aims: Development of Novel treatment strategies, including gene therapy, evaluation of the HRQL in to evaluate the potential beneficial effects of the novel treatment strategies. Study design/Methods or/PROMS: - Baseline study in all and in candidates before and after treatment paper-pencil-survey,

PBC Induced Fatigue Treated With Thiamine (PIFT)

Background/aims: Primary biliary cholangitis (PBC) is a chronic autoimmune liver disease characterised by destruction of the intrahepatic bile ducts leading to liver inflammation and fibrosis, and ultimately liver cirrhosis and end-stage liver disease. More than 50% of patients with PBC