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PSC Quality of Life Measure

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Awarded to the University College London

Dr Douglas Thorburn, Consultant Hepatologist Clinical Director For Liver Transplantation, HPB, Hepatology, Endoscopy & Gastroenterology, Royal Free Hospital/ University College London.

The total grant awarded is £50,000

Duration of award: 01 February 2017 to 31 December 2020

Award details: Developing a Quality of Life Tool for PSC

The UK-PSC Quality of Life Measure (UK-PSC-QoL)

Overview and Current Status

We are developing a Quality of Life measure for people with PSC. This is a questionnaire, and is important because people with PSC experience many quality of life and wellbeing issues, yet there is no validated way to measure them. Without a PSC-specific quality of life measure, we cannot say whether a new drug improves quality of life, or indeed makes quality of life worse.  In the absence of a reliable quality of life measure that addresses issues important to people with PSC, researchers have no choice but to use generic questionnaires, or worse, quality of life questionnaires developed for different diseases. This is not good enough.

The steering committee overseeing the entire development comprises:

Elena Marcus, Professor Douglas Thorburn (UCL/Royal Free, London), Professor David Assis (Yale), Elena Marcus, Dr Bella Vivat, Ricky Safer (PSC Partners) and Martine Walmsley (PSC Support).

PSC Support Slide 2 for PSC Forum

The questionnaire has been developed as a 3-year PhD project undertaken by University College London PhD student, Elena Marcus, under the supervision of Paddy Stone, Bella Vivat and Douglas Thorburn. The team has a strong interest in quality of life and symptom assessment. In particular, Dr Vivat is and is one of the two principal investigators on the first international project to develop a cross-cultural, multi-lingual measure of spiritual wellbeing for people receiving palliative care for cancer, on behalf of the Quality of Life Group of the European Organisation for Research and Treatment of Cancer - EORTC.  The approach taken to develop the UK-PSC Quality of Life Measure is grounded in the well-established guidelines developed by the Quality of Life Group of the EORTC with some modifications to take into account specific issues relevant to PSC.


The development of the UK-PSC Quality of Life measure comprises of 4 phases:

  1. Identification of the quality of life issues
  2. Construction of the questionnaire
  3. Pilot testing
  4. International questionnaire development
PSC Support Slide 3 for PSC Forum

Phase 1

Quality of life issues relevant to people living with PSC in the UK were identified and a provisional questionnaire was developed.

Nearly 400 issues were identified from PSC patient survey data and a systematic literature review of existing quality of life measures.

The relevance and importance of these issues were explored through interviews and group discussions with people with PSC and with experienced clinicians, and the list of issues was reduced to 89. Thank you to everyone who helped with this phase of the study.

Phase 2

Elena then constructed these issues into 83 questionnaire items for the provisional measure, the UK-PSC-QoL.

Phase 3

Elena investigated the functioning of the provisional questionnaire and its items with people living with PSC in the UK. This included looking at participants’ perceptions around acceptability, clarity, relevance and redundancy of the items and the measure. Elena is currently analysing the phase 3 (pilot-testing) UK data including looking at the acceptability of the items and their functioning.

Phase 4

Before the questionnaire can be taken forward for large scale international validation, some additional international development work will need to be undertaken. This is to ensure the questionnaire in development is robust and cross-culturally valid, and will build on the UK-based research Elena has completed as part of her PhD.

Elena will prepare the study documentation for the international development phase for each centre. The local centres will obtain local research approvals and commence with phase 1 and phase 3 work locally as described above. As with the UK development, people with PSC will be recruited across all relevant phenotypes, including pre-and post-transplant PSC and those with IBD. Local one-to-one interviews will be conducted, transcribed and translated for analysis in collaboration with Elena in the UK.

We anticipate that the international development will take around 18 months.

The final stage will involve large scale questionnaire validation across all centres involved and we expected to have 40 questionnaire items.


July 2016

We are developing a measure to assess the quality of life of people living with primary sclerosing cholangitis (PSC), under the supervision of Dr Douglas Thorburn and academics in the Marie Curie Palliative Care Research Department (MCPCRD) at University College London.

This is an exciting, ground-breaking project, aiming to produce the first validated quality of life measure for people living with PSC, with real scope to influence care and treatment.  There is a critical need for such a measure a, since PSC patients encounter many quality of life related issues, and it is important to develop treatments for PSC that improve quality of life (see recent PSC Support survey results on unmet needs and research implications). Equally, it is important that novel therapies for PSC do not have a negative effect on patients’ quality of life.

PSC Support and the British Liver Trust are therefore developing a PSC-specific quality of life measure for UK patients. The funding for this UK PhD project has been provided by the British Liver Trust, to fund a student to undertake the work in the UK and PSC Support. The initial funding became available in a bequest from Mary and Bill (pictured below) who specified that they wanted their money to be used towards an important PSC study. The principal investigator is Dr Douglas Thorburn, supported by experienced researchers from the UCL Marie Curie Palliative Care Research Department. The UK project is expected to be completed in 2018/9, and the measure may then proceed to international validation, hoping for completion by 2020/1.

Our vision for a Quality of Life measure was presented at the March 2016 AASLD/FDA Conference on Trial Design and Endpoints for Clinical Trials in Adults and Children with primary sclerosing cholangitis and has received international interest from leading researchers and key regulatory authorities (FDA and EMA). The proposed measure will be key in measuring the quality of life for people with PSC and will be suitable for multicentre international clinical trials, to evaluate patients’ responses to new treatments for PSC.

PhD candidate Elena Marcus was appointed July 2016, and began work to develop the measure in October 2016.

Martine Walmsley, Chair of Trustees for PSC Support said, 'We are delighted to have attracted a researcher of Elena’s calibre to join our team. During her interview, Elena demonstrated the passion, ability and experience necessary to ensure the delivery of a truly high-quality PSC QoL measure. This project is critical and we're excited it's moving forward.'

Mary and Bill

October 2016

PSC Partners joins the team, contributing funding towards international development with PSC Support and the British Liver Trust.

November 2017

The first stage the first phase of interviews/focus groups begins.

January 2018

Video released updating on progress of project:

October 2018

Phase 2 completed and Phase 3 began.

October 2019

Elena is now analysing data from the second stage of the study.

We now have a provisional quality of life questionnaire which is ready for international development and validation.

July 2022

After pausing due to the COVID pandemic, a description of the methodology used to develop the PSC quality of life measure was published.

Marcus E, Stone P, Thorburn D, Walmsley M, Vivat B. Quality of life (QoL) for people with primary sclerosing cholangitis (PSC): a pragmatic strategy for identifying relevant QoL issues for rare disease. Journal of Patient-Reported Outcomes. 2022 Dec;6(1):1-2.


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