Future research has the potential power to transform treatments for cystic fibrosis. Yet patients with the condition – and the people who care for them – have traditionally had little say in where studies should focus their efforts.
Now a partnership led by academics at The University of Nottingham has offered a voice to people affected by cystic fibrosis in a bid to identify the top 10 most pressing priorities for research into the condition.
Alan Smyth, Professor of Child Health & Head of Division of Child Health, Obstetrics & Gynaecology at The University of Nottingham, said: “Cystic fibrosis (CF) is an inherited condition which begins at birth but affects both children and adults. There are 10,000 people with CF in the UK, half of whom are adults. People with CF have difficulty digesting food and gaining or maintaining weight. They are also prone to recurrent and chronic chest infections.
“The James Lind Alliance Priority Setting Partnership in cystic fibrosis (CF) has brought together over 600 people from across the world and includes an even split of medical staff and lay people. Using an innovative online approach, we have collected over 1000 research ideas and have worked with the CF community to choose the 10 research questions which are the top priority to move into clinical studies.
“This is the first time such a collaborative and representative process has been undertaken in CF. We will now work with NIHR, the UK CF Trust, the US CF Foundation and the UK Research Councils to get these research questions funded and taken forward by experienced and collaborative researchers.”
Cystic fibrosis (CF) is an inherited multi-system condition that is caused by a faulty gene that causes a channel in cells to function poorly or not at all. In the lungs, this causes thick, sticky mucus to build up which often leads to infections. In other parts of the body including the gut, pancreas and liver this also causes problems.
The Cystic Fibrosis PSP is an independent group of patients and health professionals funded by The University of Nottingham, the Cystic Fibrosis Trust and Nottingham Hospitals Charity. It is being overseen and monitored by the not-for-profit organisation the James Lind Alliance, which is managed by the National Institute for Health Research (NIHR) Evaluation, Trials and Studies Co-ordinating Centre.
The priority setting exercise consisted of three phases: a survey to collect questions and research ideas from patients and health care professionals; a ranking exercise in which participants were asked to vote for their favourite topics from a list of most frequently asked treatment uncertainties; and, finally, a workshop at which the most popular treatment uncertainties were prioritised into a top 10.
The exercise had 513 individual responses to the first survey with a total of 1122 questions submitted. More than 675 people completed the second survey to select their preferred questions from a list of 71 uncertainties. Both had a 50:50 split of lay versus clinical respondents. The workshop was attended by 40 people representing the CF community, patients and health professionals, and researchers.
Speaking after the workshop CF patient Rachel Taylor said: “I found the whole event a very positive and purposeful project to be a part of and I feel we really achieved a direction and some focus for cystic fibrosis. I felt very listened to and included around the table discussions.”
The Top 10 Research Priorities for Cystic Fibrosis are:
- What are the effective ways of simplifying the treatment burden of people with CF?
- How can we relieve gastro-Intestinal (GI) symptoms, such as stomach pain, bloating and nausea in people with CF?
- What is the best treatment for non-tuberculous mycobacterium (NTM) in people with CF (including when to start and what medication)?
- Which therapies are effective in delaying or preventing progression of lung disease in early life in people with CF?
- Is there a way of preventing CF related diabetes (CFRD) in people with CF?
- What effective ways of motivation, support and technologies help people with CF improve and sustain adherence to treatment?
- Can exercise replace chest physiotherapy for people with CF?
- Which antibiotic combinations and dosing plans should be used for CF exacerbations and should antibiotic combinations be rotated?
- Is there a way of reducing the negative effects of antibiotics e.g. resistance risk and adverse symptoms in people with CF?
- What is the best way of eradicating Pseudomonas aeruginosa in people with CF?
More information about these priorities and Top 10 priorities for other conditions is available online.
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Notes to editors: The University of Nottingham has 43,000 students and is ‘the nearest Britain has to a truly global university, with a “distinct” approach to internationalisation, which rests on those full-scale campuses in China and Malaysia, as well as a large presence in its home city.’ (Times Good University Guide 2016). It is also one of the most popular universities in the UK among graduate employers and was named University of the Year for Graduate Employment in the 2017 The Times and The Sunday Times Good University Guide. It is ranked in the world’s top 75 by the QS World University Rankings 2015/16, and 8th in the UK for research power according to the Research Excellence Framework 2014. It has been voted the world’s greenest campus for four years running, according to Greenmetrics Ranking of World Universities.
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