Cystic Fibrosis Top 10

  1. What are the effective ways of simplifying the treatment burden of people with Cystic Fibrosis?
  2. How can we relieve gastro-intestinal (GI) symptoms, such as stomach pain, bloating and nausea in people with Cystic Fibrosis?
  3. What is the best treatment for non-tuberculous mycobacterium (NTM) in people with Cystic Fibrosis (including when to start and what medication)?
  4. Which therapies are effective in delaying or preventing progression of lung disease in early life in people with Cystic Fibrosis?
  5. Is there a way of preventing Cystic Fibrosis related diabetes (CFRD) in people with Cystic Fibrosis?
  6. What effective ways of motivation, support and technologies help people with Cystic Fibrosis improve and sustain adherence to treatment?
  7. Can exercise replace chest physiotherapy for people with Cystic Fibrosis?
  8. Which antibiotic combinations and dosing plans should be used for Cystic Fibrosis exacerbations and should antibiotic combinations be rotated?
  9. Is there a way of reducing the negative effects of antibiotics eg, resistance risk and adverse symptoms in people with Cystic Fibrosis?
  10. What is the best way of eradicating Pseudomonas aeruginosa in people with Cystic Fibrosis?

The following questions were also discussed and put in order of priority at the workshop:

  1. What is the best treatment plan for chronic suppression of Pseudomonas aeruginosa in people with Cystic Fibrosis?
  2. What is the most effective way of reducing lung inflammation in people with Cystic Fibrosis? 
  3. What types of psychological and family support benefit people with Cystic Fibrosis to have regular treatment and good quality of life?
  4. What is the most effective management of anxiety and depression in people with Cystic Fibrosis?
  5. What types or combinations of exercise are most beneficial for people with Cystic Fibrosis at different levels of clinical status?
  6. How do we manage an ageing population with Cystic Fibrosis?
  7. What are the long term effects of medications for Cystic Fibrosis?
  8. What is the best approach for end of life care in people with Cystic Fibrosis? 
  9. What are the long term effects of CFTR modulators, such as Ivacaftor (“Kalydeco”), (including adverse events, quality of life, Cystic Fibrosis related diabetes, reduction in medications and prevention of complications of Cystic Fibrosis)?
  10. Are there differences in outcomes when IV antibiotics are delivered at home compared with in hospital?