Cystic Fibrosis Refresh Top 10 priorities (priority setting in association with the JLA)
- What options are available for those not able to take current CFTR modulators (including rarer mutations, not eligible and unable to tolerate)?
- What is the best way to diagnose lung infection when there is no sputum e.g. children and those on modulators?
- How can we relieve gastro-intestinal symptoms, such as stomach pain, bloating and nausea?
- How do we manage an ageing population with CF?
- Is there a way of reducing the negative effects of antibiotics e.g. resistance risk and adverse symptoms in people with CF?
- What are the long-term effects of medications (including CFTR modulators) in CF?
- What are the effects of modulators on systems outside the lungs such as pancreatic function, liver disease, gastrointestinal, bone density etc?
- What are the effective ways of simplifying the treatment burden of people with CF?
- Can genetic therapies (such as gene editing, stem cell and mRNA technology) be used as a treatment for CF?
- Is there a way of preventing CF related diabetes (CFRD) in people with CF?
The following questions were also discussed and put in order of priority at the workshop:
- How can we recognise and manage the side-effects of CFTR modulators (including those at greater risk e.g. liver disease)?
- Are people with CF at higher risk of certain cancers and what is the best way to detect and manage cancers in people with CF?
- Can exercise replace chest physiotherapy in people with CF?
- How best to manage and support people with CF post-transplantation (e.g. mental health, rejection, modulator use)?
- What is the optimum treatment regimen for eradication of Pseudomonas in people with CF?
- What effective ways of motivation, support and technologies help people with CF improve and sustain adherence to treatment?
- How to improve breathing capacity/lung volume?
For full details of all of the questions identified by this PSP, please see the document below.