Final Workshop 2022

This took place online over 2 days in November. Participants were chosen from applicants to represent different aspects of the patient, clinical and research community.

17 questions shortlisted from the ranking survey were discussed and a priority order jointly agreed as follows:

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? (Ranked 2nd in 2017)

4. How do we manage an ageing population with CF? (Ranked 16th in 2017)

5. Is there a way of reducing the negative effects of antibiotics e.g. resistance risk and adverse symptoms in people with CF? (Ranked 9th in 2017)

6. What are the long-term effects of medications (including CFTR modulators) in CF? (Ranked 19th in 2017)

7. What are the effects of modulators on systems outside the lungs such as pancreatic function, liver disease, gastro-intestinal, bone density etc.?

8. What are the effective ways of simplifying the treatment burden of people with CF? (Ranked 1st in 2017)

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? (Ranked 5th in 2017)

11. How can we recognise and manage the side-effects of CFTR modulators (including those at greater risk e.g. liver disease)?

12. 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?

13. Can exercise replace chest physiotherapy in people with CF? (Ranked 7th in 2017)

14. How best to manage and support people with CF post-transplantation (e.g. mental health, rejection, modulator use)?

15. What is the optimum treatment regimen for eradication of Pseudomonas in people with CF? (Ranked 10th in 2017)

16. What effective ways of motivation, support and technologies help people with CF improve and sustain adherence to treatment? (Ranked 6th in 2017)

17. How to improve breathing capacity/ lung volume?

If you want to see all 71 questions that were formed from the new question suggestions and the ones chosen as most important still from the 2017 top 20 then they can be found here.