Cystic Fibrosis Refresh Top 10 priorities (priority setting in association with the JLA)

  1. What options are available for those not able to take current CFTR modulators (including rarer mutations, not eligible and unable to tolerate)?
  2. What is the best way to diagnose lung infection when there is no sputum e.g. children and those on modulators?
  3. How can we relieve gastro-intestinal symptoms, such as stomach pain, bloating and nausea?
  4. How do we manage an ageing population with CF?
  5. Is there a way of reducing the negative effects of antibiotics e.g. resistance risk and adverse symptoms in people with CF?
  6. What are the long-term effects of medications (including CFTR modulators) in CF?
  7. What are the effects of modulators on systems outside the lungs such as pancreatic function, liver disease, gastrointestinal, bone density etc?
  8. What are the effective ways of simplifying the treatment burden of people with CF?
  9. Can genetic therapies (such as gene editing, stem cell and mRNA technology) be used as a treatment for CF?
  10. 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:

  1. How can we recognise and manage the side-effects of CFTR modulators (including those at greater risk e.g. liver disease)?
  2. 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?
  3. Can exercise replace chest physiotherapy in people with CF?
  4. How best to manage and support people with CF post-transplantation (e.g. mental health, rejection, modulator use)?
  5. What is the optimum treatment regimen for eradication of Pseudomonas in people with CF?
  6. What effective ways of motivation, support and technologies help people with CF improve and sustain adherence to treatment?
  7. How to improve breathing capacity/lung volume?