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.
We work closely with the Cochrane CF and Genetic Disorders group of which Professor Alan Smyth is the co-ordiinating editor. Our priority setting work in CF helps to ensure that Cochrane review titles are those which are important to the CF community. Go to the website to see a list of priority titles.
What are we doing about
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:
- Antibiotic adjuvant therapy for pulmonary infection
- Antibiotic strategies for eradicating Pseudomonas aeruginosa
- Therapies for preventing recurrence of Pseudomonas aeruginosa in people with cystic fibrosis
- Digital technology for monitoring adherence to inhaled therapies and for predicting and intervening early in exacerbations
- Short-acting inhaled bronchodilators in CF
- Dosing regimens for pancreatic enzyme replacement therapy (PERT) in CF
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 long-acting inhaled bronchodilators 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
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.
In 2016-17 we undertook 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 with CF improve and sustain adherence to treatment?
- Can exercise replace chest physiotherapy?
- 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?
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.
If you want to have a look at all of the questions that were submitted they are listed here (the wording, spelling and grammar are as they were submitted to us).
James Lind CF 2
We are currently working on phase 2 of our James Lind priority setting work. 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 looked at is Q1 which is exploring the treatment burden for people with CF and their families. We sent out a survey via Twitter to help us get a better understanding of what is meant by treatment burden and followed this up with a focus group in the autumn of 2018.
We have also sent surveys out to help us answer Q2, Q6 and Q7, asking how we can relieve gastro-intestinal symptoms, about effective ways of motivation, support & technology, and whether chest physiotherapy can be repoaced with exercise. The data has been collated and refined to produce some answerable research questions,
A final survey to get the CF community's opinion on these refined questions will be coming soon…
Read our privacy notice here
Here is a short video which explains what we’re doing for phase 2
4. Understanding gut symptoms in CF
Gut investigations are currently invasive or expose patients to radiation and there is little objective evidence on the mechanisms of gut disease in CF. We aim to use Magnetic Resonance Imaging to assess the gut function and transit in CF. Currently we have completed recruitment for our observational study of people with CF and controls. We are currently open to recruitment for a CFTR modulator cross-over trial.
Ojha S, Szatkowski L, Sinha R, et alRojiroti microfinance and child nutrition: a cluster randomised trialArchives of Disease in Childhood Published Online First: 10 October 2019. doi: 10.1136/archdischild-2018-316471
Rowbotham NJ, Smith SJ, Elliott ZC, Leighton PA, Rayner OC, Morley R, et al. Adapting the James Lind Alliance priority setting process to better support patient participation: an example from cystic fibrosis. Research Involvement and Engagement. 2019;5(1):24.
Gichuru W, Ojha S, Smith S, et al. Is microfinance associated with changes in women's well-being and children's nutrition? A systematic review and meta-analysis. BMJ Open 2019;9:e023658
Nasuf AWA, Ojha S, Dorling J.Oropharyngeal colostrum in preventing mortality and morbidity in preterm infants.Cochrane Database of Systematic Reviews2018, Issue 9. Art. No.: CD011921.
Rowbotham NJ,Smith S, Prayle AP, et al. Gaps in the evidence for treatment decisions in cystic fibrosis: a systematic review. Thorax 2019;74(3):229-36 doi:10.1136/thoraxjnl-2017-210858
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
Hurley MN, Fogarty A, McKeever TM, Goss CH, Rosenfeld M, Smyth AR. Early Respiratory Bacterial Detection and Antistaphylococcal Antibiotic Prophylaxis in Young Children with Cystic Fibrosis. Ann Am Thorac Soc. 2018;15(1):42-48
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
Nicola J Rowbotham, Sally C Palser, Sherie J Smith & Alan R Smyth (2019)Infection prevention and control in cystic fibrosis: a systematic review of interventions, ExpertReview of Respiratory Medicine, 13:5, 425-434, DOI: 10.1080/17476348.2019.1595594
Cochrane CF and Genetic Disorders webpage