This feature discusses issues around cystic fibrosis and survival that some readers may find upsetting.
1. What attracted you to cystic fibrosis (CF) research?
I first became interested in CF research five or six years ago by chance, when I had the opportunity to speak with a US doctor and researcher in CF who was seeking some statistical advice. It grew gradually from there and I became involved in planning a US study. This in turn led to me making contact with CF researchers in the UK, which led to me becoming a co-investigator on the Strategic Research Centre "Cystic Fibrosis Epidemiological Network - Harnessing Data to Improve Lives".
A couple of years ago I started applying for funding to pursue my own research and I was keen to make CF a major part of this. In 2015 I was awarded a Medical Research Council Fellowship which will fund me for four years. My research has two aspects. One aspect is to apply state-of-the-art statistical methods to CF patient Registry data to answer questions about survival in cystic fibrosis. I’m focusing on the UK CF Patient Registry and the US CF Foundation Patient Registry for this. The other aspect involves the development of statistical methods for answering questions about how features of individuals impact on their survival.
I hope that CF is going to continue to be a major part of my research in the future. My aim is to develop a team of researchers working in CF using patient Registry data.
2. Your work focuses on life expectancy, which seems like a pretty challenging subject. Why did you decide to focus on this?
The fact that CF is a life-shortening condition, and that it affects young people, I feel makes 'survival' such a fundamental issue for people with CF, which makes it a really important target for research. From discussions with patients and doctors, and from the research literature, it became clear to me that there is still a lot to learn about what impacts on survival in cystic fibrosis. Given that survival of people with CF has improved so much over recent decades, there is also much to be done in terms of providing general information on life expectancy that is as up-to-date as possible. Furthermore, I believe there is a need to improve the way in which information is presented to people with cystic fibrosis. As part of my research I am particularly keen on presenting information on survival in CF to patients in an understandable and accessible way.
Much of my previous research outside of CF had involved study of survival and I also teach statistical methods for investigating factors related to survival, so this is one of my main areas of expertise.
3. How do you find getting people to talk about life expectancy? It must be a rather personal or emotive issue.
I have spoken to people in an informal way about life expectancy, as we all probably do at some point. But this questionnaire is the first time I am trying to collect more detailed information in a formal way on thoughts about life expectancy from people with cystic fibrosis. I have been very conscious of wanting the questionnaire to approach the topic in a sensitive way and am really grateful for input on the content and wording of the questionnaire from patient representatives as well as from a clinician with many years of experience of working with people with CF and a Cystic Fibrosis Trust representative. I hope that people who complete the questionnaire will find that the topic is approached sensitively. My impression from speaking with people with CF is that they can be very stoic and matter-of-fact when it comes to discussing life expectancy, which I imagine comes from CF being something they have always lived with. Because the questionnaire is anonymous I hope that people will feel able to say what they are really feeling and thinking.
4. What are you looking to achieve with the survival survey? What will you do with the results?
I believe this will be the first questionnaire that tries to collect information on this topic from people with cystic fibrosis The overall aims are to understand better the extent to which people with CF would like to know about their life expectancy, how people with CF discover and use information about their life expectancy and what people with CF might like to know about life expectancy, and time to other outcomes, which is currently not available to them.
The results will provide information about the degree to which people with CF are interested in more personalised information about their life expectancy and insights into the kinds of outcomes, in addition to survival, that people with CF are interested in, for example the expected age at which they might have a transplant or reach a certain level of lung function.
I will build the responses from the questionnaire into my research so that the statistical prediction models that I develop will address the questions that patients are most interested in. The questionnaire asks about the format and environment in which patients would like information about survival and other outcomes to be presented to them. I will incorporate this information into my plans for how the results from statistical prediction models are presented.
A summary of the results from the questionnaire will be published later this year as an information sheet. This will be advertised by the Cystic Fibrosis Trust and will appear on my website. People can also provide their email address in the questionnaire so that they can receive the information sheet by email when it is ready. I will also write the results up for publication in an academic journal, which will reach other CF researchers and doctors and therefore be informative for the work that other people are doing in CF, both directly with patients and via research.
5. How will what you learn have an impact on people living with cystic fibrosis? How do you think it will impact on their families and friends too?
The immediate results from the questionnaire will, I hope, be informative and interesting for people with CF because they will provide a summary of the kinds of issues that people with CF are thinking about with regard to their life expectancy and other health outcomes.
I will use the responses to the questionnaire to inform the direction of my research, so the questionnaire will directly impact the type of information that I develop and also the way in which it is presented for patients. Ultimately this will enhance the information that is available to people with CF, and to their care teams, families and friends. In particular, people will ultimately be able to access more personalised information about their future prospects in terms of survival and other events that may occur, which takes into account their individual health status.
The survey is open to anyone with cystic fibrosis over 16 years of age. If you would like to speak to someone about any of the difficult issues addressed in this feature or the survey itself, you can contact the Cystic Fibrosis Trust helpline.