Currently, we are targeting evidence synthesis in the field of Cystic Fibrosis (CF), but we will expand into other areas of Paediatrics as our group grows.
What we are currently working on
1. Assessing the evidence base to inform decision making in clinical care
A systematic review attempts to identify, appraise and synthesise all the empirical evidence that meets pre-specified eligibility criteria to answer a given research question.
To do this we use explicit methods aimed at minimising bias, in order to produce more reliable findings that can be used to inform decision making. At present the following Cochrane systematic reviews are being undertaken within Evidence Based Child Health:
- Long-acting inhaled bronchodilators for cystic fibrosis
- Inhaled antibiotics for long-term therapy in cystic fibrosis
- Oto-protective strategies to prevent drug-induced hearing loss in infectious diseases
- Therapies for preventing recurrence of Pseudomonas aeruginosa in people with cystic fibrosis
Members of the group are also authors on other reviews which are going through the routine update cycle, including percutaneous lines for intravenous antibiotics in cystic fibrosis, intravenous antibiotics in cystic fibrosis, and antibiotic adjuncts in cystic fibrosis.
Prof Alan Smyth is coordinating editor of the Cochrane Cystic Fibrosis and Genetic Disorders Group.
2.Identifying evidence gaps in treatment decisions in CF
We do not always have the evidence to support treatment decisions. We are conducting a systematic review to identify the known evidence gaps in treatment decisions in CF. These evidence gaps will be used to guide both researchers and funding bodies to priority areas.
3.Shaping the future research agenda in partnership with the CF community
Presently there is no cohesive strategy to fill the above-mentioned evidence gaps, with research being driven by financial interests or those of investigators. Traditionally patient views have not been considered when setting research agendas. In partnership with the CF community we have collected and prioritised these uncertainties for future research questions.
We have undertaken a James Lind Alliance Priority Setting Partnership (JLA PSP) in CF. This brought together patients, families, healthcare professionals and commissioners to agree shared priorities for research.
Top 10 priorities for clinical research in CF
Here are our final top ten questions as agreed by the CF community:
- What are the effective ways of simplifying the treatment burden of people with CF?
- How can we relieve gastro-intestinal symptoms, such as stomach pain, bloating and nausea?
- What is the best treatment for non-tuberculous mycobacterium (including when to start and what medication)?
- Which therapies are effective in delaying or preventing progression of lung disease in early life?
- Is there a way of preventing CF related diabetes?
- What effective ways of motivation, support and technologies help people withCF improve and sustain adherence to treatment?
- Can exercise replace chest physiotherapy?
- Which antibiotic combinations and dosing plans should be used forCF 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?
We have addressed the challenges of infection control by running the PSP steering group (and also our outreach to the CF community) through web-based conferencing systems.
More information about the PSP.
James Lind CF 2
In phase 2 we will be taking four of the top ten priorities which are broad in their nature. We want to delve a bit deeper to find out what the CF community understand by them and at the same time to try and identify more specific questions that can be answered by research.
We will be reaching out to the whole CF community via online surveys and focus groups. We will be making use of videoconferencing so that we can include as many people as possible without cross-infection risks.
The four questions we will be focussing on are:
The first question we will be looking at is Q1 which is exploring the treatment burden for people with CF and their families.
The first online survey will be open to everyone on the 21st March 2018
Here is a short video which explains what we’re doing for phase 2
4.Communicating research to the patient community
We set up the CF Unite project
which uses a bespoke online platform, developed by our team, in conjunction with The University of Nottingham. This allows the patient community to choose a topic and participate in online events where scientists present their work and answer questions. Previous topics have included gene therapy and new drugs to overcome the CF defect. The site also publishes lay summaries of CF research papers.
5.Understanding the blocks to evidence medicine
There are a number of blocks in the path from evidence to change in clinical practice. We are engaged in a number of studies to systematically study these blocks. We have found that in the field of cystic fibrosis, up to half of registered clinical trials are not published within 5 years of trial completion.
Our studies on trial registration and trial summary level data publication have informed UK and international policy development in the area of trial publication and dissemination regulations. Our research has been used by important international campaigns, such as the AllTrials.net.
Blocks to incorporating the best evidence in clinical trials
When a health professional prescribes a treatment, they use guidelines to help decide which treatments to recommend. These guidelines should be informed by the best available evidence. The Cochrane collaboration are well known for producing high quality clinical evidence which can inform guidelines – they write Cochrane Reviews which systematically look for all the available evidence on a narrow topic and then judge if a treatment works or not.
We studied all the United Kingdom guidelines for children’s respiratory disease, and found that some or all of the relevant Cochrane reviews were not used in preparing guideline recommendations 40% of the time.
We have made an interactive figure to illustrate our data, and you can also read the open access paper.
Rowbotham NR, Smith S, Leighton P et al (2018) The top 10 research priorities in cystic fibrosis developed by a partnership between people with CF and healthcare providers. Thorax 73(4): 388-90 DOI: 10.1136/thoraxjnl-2017-210473
Kwok TC, Ojha S, Dorling J (2017) Feed thickener for infants up to six months of age with gastro-oesophageal reflux. Cochrane Database of Systematic Reviews DOI: 10.1002/14651858.CD003211.pub2
Prayle AP, Cox T, Smith SJ, Rycroft-Malone J, Thomas KS, Hughes DA, Smyth AR. (2017) Do guidelines for treating chest disease in children use Cochrane Reviews effectively? A systematic review. Thorax doi: 10.1136/thoraxjnl-2016-208790. [Epub ahead of print]
Rowbotham, Nicola J. & Smyth, Alan R. (2017) The patient voice in research — Supporting actor or starring role? Journal of Cystic Fibrosis DOI: http://dx.doi.org/10.1016/j.jcf.2017.03.001
Smith S, Edwards CT (2017) Long-acting inhaled bronchodilators for cystic fibrosis. Cochrane Database of Systematic Reviews DOI: 10.1002/14651858.CD012102.pub2
Smyth AR, Walters S (2017) Prophylactic anti-staphylococcal antibiotics for cystic fibrosis. Cochrane Database of Systematic Reviews DOI:10.1002/14651858.CD001912.pub4
Jain, K., et al. (2016) Bronchoscopy-guided antimicrobial therapy for cystic fibrosis. Cochrane Database of Systematic Reviews DOI: 10.1002/14651858.CD009530.pub3
Lo David, K. H., et al. (2015) Interventions for the eradication of meticillin-resistant Staphylococcus aureus (MRSA) in people with cystic fibrosis. Cochrane Database of Systematic Reviews DOI: 10.1002/14651858.CD009650.pub3
Langton Hewer Simon, C. and R. Smyth Alan (2014) Antibiotic strategies for eradicating Pseudomonas aeruginosa in people with cystic fibrosis. Cochrane Database of Systematic Reviews DOI: 10.1002/14651858.CD004197.pub4
Smyth Alan, R. and J. Bhatt (2014) Once-daily versus multiple-daily dosing with intravenous aminoglycosides for cystic fibrosis. Cochrane Database of Systematic Reviews DOI: 10.1002/14651858.CD002009.pub5
Smyth Alan, R. and S. Walters (2014) Prophylactic anti-staphylococcal antibiotics for cystic fibrosis. Cochrane Database of Systematic Reviews DOI: 10.1002/14651858.CD001912.pub3
Hurley Matthew, N., et al. (2013) Antibiotic adjuvant therapy for pulmonary infection in cystic fibrosis. Cochrane Database of Systematic Reviews DOI: 10.1002/14651858.CD008037.pub3
Hurley M, Prayle A, Flume P. Intravenous antibiotics for pulmonary exacerbations in people with cystic ﬁbrosis. Cochrane Database Syst Rev 2012(3):CD009730
Hurley MN, Prayle AP, Smyth AR. Delayed publication of clinical trials in cystic fibrosis. J Cyst Fibros 2012;11(1):14-7 doi: S1569-1993(11)00137-8 [pii]
Prayle AP, Hurley MN, Smyth AR. Compliance with mandatory reporting of clinical trial results on ClinicalTrials.gov: cross sectional study. BMJ 2012;344:d7373
Prayle Andrew, P., et al. (2010) Percutaneous lines for delivering intravenous antibiotics in people with cystic fibrosis. Cochrane Database of Systematic Reviews DOI: 10.1002/14651858.CD008243.pub2