These are the new unanswered questions formed from suggestions given from the first survey in our 2022 refresh, plus the questions voted as most important from our 2017 priority setting exercise. There are 71 in total and we have tried to group them in similar themes but there may fit in more than one.
More details around each of these and the evidence checks that occurred will shortly be available on the JLA website.
Burden & adherence
What effective ways of motivation, support and technologies help people with CF improve and sustain adherence to treatment?
What are the effective ways of simplifying the treatment burden of people with CF?
How can we support self-care and adherence to treatment in specific groups with CF such as children, adolescents and the digitally excluded?
Ageing
How do we manage an ageing population with CF?
How can we promote good mental health in people with CF as they grow older?
Bones & joints
What is the optimum management for CF related arthritis (joint pain) in people with CF?
How can we promote good bone health and posture in people with CF?
CFTR
What are the long-term effects of CFTR modulators?
What are the effects of modulators on systems outside the lungs such as pancreatic function, liver disease, gastrointestinal, bone density etc?
Are options available for those not able to take current CFTR modulators (including rarer mutations, not eligible and unable to tolerate)?
How can we recognise and manage the side-effects of CFTR modulators (including those at greater risk e.g. liver disease)?
What is the optimum dosing, timing and type of CFTR modulators including when to start and how to monitor?
Can genetic therapies (such as gene editing, stem cell and mRNA technology) be used as a treatment for CF?
Are the comorbidities associated with CF caused by the faulty CFTR gene?
Cancer
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?
Diet & Nutrition
What is the optimal nutritional management for people with CF (including behavioural and psychological aspects)?
How can overweight and obesity be effectively managed in people with CF?
Are dietary supplements, such as salt, fatty acids and probiotics, effective in improving symptoms of CF?
CF related diabetes
How do we effectively diagnose and manage CF-related diabetes- including the impact of exercise and CFTR modulators?
Is there a way of preventing CF related diabetes (CFRD) in people with CF?
ENT
What is the optimum management regimen for sinus disease in people with CF?
How can we prevent and treat nasal polyps in CF?
Epidemiology
What environmental factors influence health outcomes for people with CF and how can their effects be mitigated/offset? (Pollutants, allergens, toxins)
What are the implications of co-infection of Pseudomonas aeruginosa and Staphylococcus aureus for people with CF?
How does CF interact with other inherited conditions?
Can we predict or influence individual disease trajectory and exacerbation risk?
Why are some CFTR genetic mutations more severe than others and why are people with the same mutation affected differently?
Medication
How can we best manage medication interactions (both good and bad) in CF?
What are the long-term effects of medications for CF?
Does CF or its associated treatments affect the brain and/or behaviour?
Is UV effective for sanitising nebuliser equipment?
Other
What is the most effective way of reducing inflammation in people with CF?
How common are chronic pain and fatigue in CF and how should these problems be managed?
What is the role of complementary therapies in the management of CF?
How to manage lifestyle factors important for people with CF (such as sleep and stress)?
Can telemedicine (remote monitoring and care) improve outcomes for people with CF?
Gastro-intestinal
What is the most effective treatment for poor absorption and/or faltering growth?
Does anti-reflux medication prevent progression of lung disease in people with CF?
How can we relieve gastro-intestinal symptoms, such as stomach pain, bloating and nausea?
What is the optimum management of meconium ileus in neonates to improve long term prognosis?
What is the optimum regimen for pancreatic replacement therapy in people with CF?
How can we best prevent, detect and manage CF-related liver disease?
Infection & antibiotics
What is the role of therapies other than conventional antibiotics in the treatment of infections in CF? e.g. vitamins, colloidal silver, NAC, nitric oxide, bacteriophages?
What are effective ways of preventing cross-infection and/or spread of infection among people with CF and does this advice change with CFTR modulator therapy?
What alternative methods of administering antibiotics (e.g. oral/inhaled) can replace the need for IV antibiotic treatment?
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 to identify and treat existing and emerging CF pathogens including antibiotic resistant infections?
What are the effects of vaccinations (e.g. flu & COVID) in people with CF, including long term effects?
What is the optimum treatment regimen for Burkholderia?
What is the optimum treatment regimen for NTM in people with CF (including when to start and what medication)?
What is the role of Aspergillus in causing lung infection in CF and how should this be diagnosed and treated?
How best to treat Staphylococcus infection in the lungs of people with CF?
What is the optimum treatment regimen for eradication of Pseudomonas in people with CF?
Fertility & hormones
How can we best detect, monitor and treat infertility and subfertility in people with CF, including any modulator-mediated effects?
What is the optimum management of pregnancy in CF (including whether to breastfeed and which medications can still be taken or need to be stopped)?
What are the effects of female hormones on CF symptoms and progression?
How can we best detect and treat menopausal symptoms in people with CF?
Physiotherapy
Can exercise replace chest physiotherapy in people with CF?
What is the optimum physiotherapy routine for maintaining the health of people with CF with different severities of lung disease (this would include inhaled therapy, exercise, and Airway Clearance Techniques)?
Psychological
What forms of psychological support help with the mental health and wellbeing of people with CF and their families at all stages in life?
What effects do CF treatments have on mental health and can we minimise negative effects?
What are the best ways to support people with CF on their journey through parenthood?
What is the most effective management of anxiety and depression in people with CF?
Respiratory
When is the optimum time to start oxygen therapy?
What is the best way to diagnose lung infection when there is no sputum e.g. children and those on modulators?
How to eliminate or reduce atelectasis?
How to improve breathing capacity/lung volume?
What is the optimum management of haemoptysis in people with CF?
How best to manage those with end stage lung disease that are not eligible for transplant?
Which therapies are effective in delaying or preventing progression of lung disease in early life in people with CF?
Transplant
How best to manage and support people with CF post-transplantation (e.g. mental health, rejection, modulator use)?