UK-PSC Quality of Life Update

We are developing a Quality of Life measure for people with PSC.

What is a quality of life measure?

This is a questionnaire which can measure quality of life. It is sometimes called a quality of life tool or questionnaire.

Why is that important in PSC?

It is important to have a good quality of life measure because people with PSC experience many quality of life and wellbeing issues, yet there is no validated way to measure them. That means we are not asking PSC patients the right questions when it comes to talking about how day to day life is affected by PSC.

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 currently have no choice but to use generic questionnaires, or worse, quality of life questionnaires developed for different diseases.

This is not good enough.

What is PSC Support doing about it?

In 2016 PSC Support started talking to experts to develop the a disease-specific quality of life measure for PSC. We assembled a team of experts and began work!

How will it be used?

Our intention is that the UK-PSC Quality of Life Measure will be used by doctors to measure how a patient's quality of life is changing over time. This might help inform medical decision-making and treatment options. We would also like to use the measure in research to compare quality of life between patients and to evaluate the impact of clinical trial drugs and interventions. A great deal of work is necessary to develop such a measure, but we're working on it:

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

It is important to note that this study has been funded entirely by patients. We are working with PSC Partners and the British Liver Trust to deliver this important quality of life measure for PSC patients.

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.