Paeds · respiratory-sleep-and-airway
Cystic fibrosis: pulmonary and multidisciplinary management
Also known as CF lung disease management · Cystic fibrosis airway clearance and antibiotics · CFTR modulator therapy in children · Multidisciplinary cystic fibrosis care · Paediatric CF pulmonary care
Fellowship guide to the pulmonary and multidisciplinary management of cystic fibrosis in children: the CFTR channel defect that dehydrates the airway surface and starts a self-perpetuating cycle of mucus plugging, chronic infection and neutrophilic inflammation ending in bronchiectasis; the four pillars of daily airway clearance physiotherapy, mucoactive nebulisers such as dornase alfa and hypertonic saline, an anti-infective ladder from Pseudomonas eradication through cycled inhaled antibiotics and chronic azithromycin to intravenous therapy for exacerbations, and CFTR modulator therapy with elexacaftor-tezacaftor-ivacaftor; the nutrition, pancreatic enzyme and psychosocial work of the multidisciplinary team; infection segregation; recognising exacerbations, haemoptysis, pneumothorax, allergic bronchopulmonary aspergillosis and CF-related diabetes; and the ANZ, UK and North American approach to monitoring, transition and lung transplantation.
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Target exams
Red flags
- Increased cough, sputum volume or purulence, breathlessness, reduced appetite, weight loss or a drop in FEV1 — a pulmonary exacerbation that needs prompt airway clearance intensification and antibiotics, not watchful waiting
- A first isolate of Pseudomonas aeruginosa from an airway culture — attempt eradication immediately, because chronic Pseudomonas infection accelerates lung decline
- Massive haemoptysis or sudden pleuritic chest pain and breathlessness — think a bleeding bronchial artery or a pneumothorax, both of which are life-threatening emergencies
- Wheeze, a rising IgE, new infiltrates and Aspergillus positivity — suspect allergic bronchopulmonary aspergillosis, which needs corticosteroids and antifungal therapy rather than antibiotics alone
- Isolation of Burkholderia cepacia complex or non-tuberculous mycobacteria — organisms that carry a worse prognosis, demand strict segregation and can jeopardise transplant candidacy
Life stages
Care settings
Clinical exam formats
Board mappings
- General and Community Paediatrics
- Chronic respiratory disease
- Cystic fibrosis and suppurative lung disease
- Current 2026 PREP curriculum — Learning goal 5: Clinical assessment – essential general paediatrics
- Renewed curriculum for first-year trainees from 2027 — Learning goal 8: Chronic and complex care
- Paediatric respiratory medicine and multidisciplinary chronic disease competencies
- Clinical Applications
- Chronic respiratory disease and its long-term management
- Long Cases
- Short Cases
- Chronic disease and multisystem assessment scenarios
- 4. Professional skills and knowledge: Patient management
- 8. Complex and chronic conditions
- Respiratory: Manages cystic fibrosis and chronic suppurative lung disease
- Foundation of Practice (FOP)
- Applied Knowledge in Practice (AKP)
- Chronic respiratory disease; cystic fibrosis
- Clinical
- History and management planning
- Chronic respiratory assessment and the multidisciplinary plan
- General Pediatrics Content Outline — Domain 17: Respiratory System
- General Pediatrics Content Outline — Domain 9: Genetics and Dysmorphology
- General Pediatrics Content Outline — Domain 14: Nutrition
- Patient Care 1: History and Physical Examination
- Patient Care 4: Clinical Reasoning
- Patient Care 6: Chronic and complex care coordination
- Medical Knowledge 1: Clinical Knowledge
- Systems-Based Practice: multidisciplinary and high-value care
- Medical Expert
- Communicator
- Collaborator
- Health Advocate
- Pediatrics: Core EPA — Manage children with chronic complex respiratory disease within a multidisciplinary team
Overview & Definition
Picture the ten-year-old seen in the CF clinic: she is thin despite a big appetite, she has a loose productive cough most mornings, her last airway culture grew Pseudomonas, and her spirometry has slipped a little since last visit. Around her sits a team — a physiotherapist checking her airway clearance technique, a dietitian adjusting her enzymes and calories, a nurse reviewing her nebulisers, and a doctor deciding whether she is having an exacerbation. That scene is the whole of cystic fibrosis management: a chronic, multisystem disease held in check by daily preventive work rather than cured by any single treatment. [8] [9]
Cystic fibrosis is an autosomal recessive disease caused by mutations in the CFTR gene, whose protein is a chloride and bicarbonate channel at the surface of epithelial cells. When that channel fails, the fluid lining the airway becomes dehydrated, mucus becomes thick and sticky, and the lung loses its ability to clear inhaled organisms — so the pulmonary problem is fundamentally one of impaired mucociliary clearance leading to chronic infection. [9] [1]
Why this matters at fellowship level is that cystic fibrosis has been transformed within a generation. Median survival has risen from early childhood decades ago to the fifth decade and beyond, driven first by aggressive airway clearance, nutrition and infection control, and now by CFTR modulator drugs that correct the basic defect. The examiner wants a candidate who understands the disease mechanism, can structure the preventive management, and can recognise the complications and the emergencies. [8] [1]
References11ShowHide
- [1]Middleton PG, Mall MA, Dřevínek P, et al. Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele. N Engl J Med, 2019.PMID 31697873
- [2]Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med, 2011.PMID 22047557
- [3]Wainwright CE, Elborn JS, Ramsey BW, et al. Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR. N Engl J Med, 2015.PMID 25981758
- [4]Fuchs HJ, Borowitz DS, Christiansen DH, et al. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group. N Engl J Med, 1994.PMID 7503821
- [5]Elkins MR, Robinson M, Rose BR, et al. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. N Engl J Med, 2006.PMID 16421364
- [6]Saiman L, Marshall BC, Mayer-Hamblett N, et al. Azithromycin in patients with cystic fibrosis chronically infected with Pseudomonas aeruginosa: a randomized controlled trial. JAMA, 2003.PMID 14519709
- [7]Ramsey BW, Pepe MS, Quan JM, et al. Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. Cystic Fibrosis Inhaled Tobramycin Study Group. N Engl J Med, 1999.PMID 9878641
- [8]Mogayzel PJ Jr, Naureckas ET, Robinson KA, et al. Cystic fibrosis pulmonary guidelines. Chronic medications for maintenance of lung health. Am J Respir Crit Care Med, 2013.PMID 23540878
- [9]Farrell PM, White TB, Ren CL, et al. Diagnosis of Cystic Fibrosis: Consensus Guidelines from the Cystic Fibrosis Foundation. J Pediatr, 2017.PMID 28129811
- [10]Zemanick ET, Taylor-Cousar JL, Davies J, et al. A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele. Am J Respir Crit Care Med, 2021.PMID 33734030
- [11]Treggiari MM, Retsch-Bogart G, Mayer-Hamblett N, et al. Comparative efficacy and safety of 4 randomized regimens to treat early Pseudomonas aeruginosa infection in children with cystic fibrosis. Arch Pediatr Adolesc Med, 2011.PMID 21893650