Patient reported outcomes to measure treatment success
- Title: Establishing the content coverage of available Patient Reported Outcome (PRO) measures used in neuro-oncology and provide recommendations on the use of these PRO measures: part of the Response Assessment in Neuro-Oncology – Patient Reported Outcome (RANO-PRO) initiative
- Lead researcher: Professor Martin Taphoorn
- Where: Leiden University Medical Centre, Netherlands
- When: June 2019 – December 2021
- Cost: £139,273 over two years
- Research type: Patient Reported Outcomes, Quality of Life, All tumours, Academic and Clinical
- Grant round: Quality of Life
What is it?
In clinical trials and practice for adult with brain tumours, several outcomes are assessed to determine how well a treatment is working. These outcomes can include the length of survival, response of the tumour on imaging, but also the person’s abilities and wellbeing. Wellbeing reflects the person’s lived experience and is assessed using patient reported outcome (PRO) measures.
Findings from these PROs are essential for clinical decision-making, i.e. determining which treatment is best for an individual patient, or monitoring the effect of a treatment over time. Several PROs are available for use in brain tumours, but with the development of new treatment strategies, these questionnaires may no longer be completely relevant (e.g. new side effects of a treatment may not be covered). To enable the best assessment of how the treatment impacts on the individual all relevant aspects should be included in the PRO measures.
To improve the quality of PRO assessment in brain tumours, an international working group has been established. This group includes healthcare professionals from the varied disciplines that are involved in the care of people with brain tumours. The group is known as Response Assessment in Neuro-Oncology – Patient Reported Outcome initiative, or RANO-PRO.
This research grant will support the RANO-PRO team to determine if questionnaires currently used during brain tumour treatment cover all aspects that are relevant for the people receiving the treatments.
How they’ll do it
Part 1: A review of the scientific literature will be performed to identify all PRO measures that have been used in brain tumour research up to now. The focus will be on questionnaires assessing symptoms, function or health-related quality of life in people with a glioma, a primary central nervous system (CNS) lymphoma, a meningioma or brain metastases.
Part 2: In addition, the team will identify aspects of functioning and wellbeing that are most important to people with brain tumours using an international survey. These people, their informal caregivers and experts in the field of neuro-oncology will be asked to indicate which activities are most important to them.
Part 3: Next, they’ll evaluate if the currently existing questionnaires cover these areas.
Why is it important?
Based on the results of this project, the best PRO assessment will be defined. This will benefit people undergoing treatment for brain tumours through better assessment of patient-centred outcomes.
Additionally, this will result in higher quality information, which can be used for clinical decision-making and monitoring during treatment.
Who will it help?
Due to the international nature of project, with collaborators from all over the world, this project will benefit people both in the UK and other countries.
The recommendations about the existing PRO measures will help researchers to select those measures that best capture the areas that are most important to people, both in clinical trials and clinical practice.
Over time, the results of this project may further increase the benefit for people with brain tumours, as existing PRO measures can be further improved, or new relevant measures be developed.
Better assessment of patient-centred outcomes will result in higher quality information that can be used for drug approval procedures, clinical decision-making and monitoring in clinical practice.
Part 1: literature review: The team have performed an extensive literature search to identify brain tumour studies which used a PRO measure. In addition, they searched on a website where clinical trials are registered for additional studies that may have included relevant PRO measures. We identified >550 articles describing the use of a PRO measure in people with a brain tumour, and >200 studies on the website. These sources resulted in the identification of about 200 different PRO measures. Information on the studies and the PRO measures have been summarised and will be shared with the research community.
Part 2: international survey: The survey has been created including areas that could possibly be relevant for people with brain tumours and the team have submitted this, and their plan to use the results, for ethical approval at the institutions involved.
The survey will allow them to identify which areas are most important to the brain tumour community, and whether they’re covered by the existing PRO measures
Part 1: The team plan to publish a report on the literature review, showing the PRO measures that are currently being used in brain tumour studies.
Part 2: They also plan to finish data collection using the international survey.
Part 3: Based on the data obtained with the literature review and the survey, they’ll be able to evaluate if existing PRO measures cover all aspects that are relevant, or if they lack certain aspects.
Research is just one other way your regular gift can make a difference
Research is the only way we will discover kinder, more effective treatments and, ultimately, stamp out brain tumours – for good! However, brain tumours are complex and research in to them takes a great deal of time and money.
Across the UK, over 100,000 families are facing the overwhelming diagnosis of a brain tumour and it is only through the generosity of people like you can we continue to help them.
But, by setting up a regular gift – as little as £2 per month – you can ensure that families no longer face this destructive disease.
Professor Martin Taphoorn
Professor Taphoorn is both an academic and a clinician. He is a Consultant in Neuro-Oncology at the IKMN Leiden and Professor of Neuro-Oncology at the Leiden University Medical Centre