Print This PostMy name is Victoria, and I’m an early career researcher at the Bristol Brain Centre in Southmead Hospital. Over the coming months, I’ll be sharing regular blog posts on our project that aims to influence which research outcomes clinical researchers include in their studies involving individuals with mild cognitive impairment (MCI). We’ll do that by spending the next year developing a ‘core outcome set’.
In today’s introductory blog, I’ll answer two key questions: what is a core outcome set, and why do we need one for MCI research?
What is a core outcome set, anyway?
Both clinical research and clinical practice aim to improve the lives of people living with a specific condition by providing treatments or care pathways that are safe and effective. We find out if something is safe and effective by measuring “outcomes”, which we hope will improve after treatment.
Typically, a small number of researchers, clinicians, and/or funding agencies decide what constitutes the primary outcome or endpoint in a study. There are a few problems with this approach:
- We don’t know if these outcomes are the most meaningful and important for patients;
- Researchers might be tempted (or feel pressured) to measure lots of outcomes and selectively report those which support their intervention;
- It results in heterogenous outcomes being reported across different studies, preventing comparisons across studies (such as in a systematic review).
Enter the core outcome set (COS). COS are an agreed minimum group of outcomes that are recommended to be measured for a specific health condition. Ideally, COS are developed in collaboration with people with lived experience of the health condition (patients and their loved ones) and those who’ll be using it (e.g., researchers and healthcare professionals). Researchers are encouraged to report the COS as a minimum dataset of the most important outcomes that stakeholders are interested in and add in any extra outcomes relevant to their research questions. It’s designed to reduce research waste, rather than be restrictive.
Why do we need a COS in MCI?
In research involving people with cognitive problems such as MCI or dementia, we might automatically (and sensibly) look to measure cognitive outcomes, such as a score on a cognitive test or rate of progression to dementia. Deciding on the most appropriate outcome(s) is crucial – whether your outcome demonstrates a significant change will determine if the intervention is considered successful. With many interventional studies (including anti-amyloid trials) moving to include patients at the MCI stage or even earlier, choosing what would constitute a successful trial is really important.
Whilst there are several COS for dementia either established or in development, there isn’t one for MCI. Though generally considered as prodromal dementia, MCI is a syndrome characterised as cognitive impairment without significantly affecting activities of daily living. MCI can be caused by the same neurodegeneration which causes dementia, such as Alzheimer’s disease or Parkinson’s disease, or might result from other health conditions or have an unknown cause, and it doesn’t always progress into dementia.
MCI tends to affect people at a younger age than dementia and presents with different symptoms and an uncertain prognosis – we don’t know if or when they’ll develop dementia. So, we think that people with MCI represent a unique patient group who should have their own COS.
As co-chief investigator Professor Liz Coulthard explains:
“When we were designing studies for people with MCI, it was hard to work out what we should measure. We wanted to develop interventions to improve sleep that helped the things that were important to people with MCI. While it will always be important to understand how a treatment affects disease biology, it is also important to find out how it makes the patient feel. For other diseases, we know that a treatment might work really well biologically, but if it leaves people feeling very tired or unwell for a long time, it might not be worth it for people. As MCI is a relatively recently characterised diagnostic stage, there was relatively little information on what is actually important to people with MCI. We know what is important in dementia, but MCI is an earlier stage when people are independent and might be working or, for example, looking after grandchildren.”
So, over the next year, we’ll be developing a COS for people living with MCI – and putting those with lived and professional experience at the heart of it. We want to share the journey with other researchers – particularly as we hope many of you will take part, either as a participant sharing your thoughts or implementing the COS when designing your future research. So, watch this space – and feel free to get in touch!
Victoria Gabb
Author
Victoria Gabb is a Senior Research Associate in Sleep & Dementia Neuroscience at University of Bristol. Victoria is working on her PhD through publication and she is currently working on an important project to develop ‘Core Outcomes’ for Mild Cognitive Impairment. Her blogs on dementia researcher will be a way of sharing the progress of her work, to improve transparency, share the process and as a way to involve others – sharing the work, the process, and life in the pursuit of research.
