Synopsis
Coordinate specialist multisystem care through transition and adulthood, recognise pulmonary and gastrointestinal emergencies, individualise airway and nutritional treatment, and use CFTR modulators safely by genotype.
- Cystic fibrosis is an autosomal-recessive CFTR disorder affecting airway mucus, pancreas, gut, liver, sinuses, sweat and reproduction; modern modulator therapy changes but does not erase multisystem surveillance.
- Adolescents should move through a planned, developmentally appropriate transition to a specialist adult CF centre, with increasing autonomy, medicines competence and attention to education, work and mental health.
- A late diagnosis remains possible in adults with bronchiectasis, recurrent pancreatitis, malabsorption, male infertility or characteristic organisms; use sweat chloride and CFTR testing through a specialist pathway.
- Respiratory treatment combines individual airway clearance, exercise, mucoactive therapy and culture-led antimicrobial plans; do not copy non-CF bronchiectasis regimens into CF without the centre's protocol.
- CFTR modulators are genotype and product specific. NICE TA988 recommends elexacaftor–tezacaftor–ivacaftor plus ivacaftor for eligible people with at least one F508del mutation within the marketing authorisation.
- Pancreatic enzyme replacement is taken with fat-containing food and adjusted by symptoms, growth or weight and specialist dietetic review; excessive dosing is unsafe.
- Screen proactively for CF-related diabetes, liver disease, low bone density, kidney toxicity, nutritional deficiency, fertility needs and treatment-related anxiety or depression.
- People with CF should not be placed together casually in clinics or wards because cross-infection with transmissible bacteria and NTM remains a material safety concern.
Key red flags
A large fresh bleed, sudden unilateral pleuritic pain, marked desaturation or acute respiratory distress requires immediate imaging, airway assessment and CF-respiratory escalation; suspend forceful clearance until reviewed.
Colicky right-lower-quadrant pain, distension, reduced stool and a palpable caecal mass suggest partial DIOS; bilious vomiting, peritonism or complete obstruction requires surgical and CF gastroenterology assessment.
Investigation priorities
Assess CFTR dysfunction in a person with a compatible phenotype or newborn-screen follow-up.
Management branches
An adolescent is developing the capacity to manage treatment and prepare for adult services.
- Begin planning over several years, assess understanding and practical skills, include private consultation time, and agree how parents or carers remain supportive without displacing the young person's voice.
- Transfer a complete summary of genotype, best lung function, microbiology, allergies, venous access, complications, modulator history, fertility discussion, mental health and emergency plans to the adult centre.
- Address school or university, employment, benefits, relationships, contraception, pregnancy and adherence openly; confirm the first adult appointment before paediatric follow-up closes.
Key medicines
Elexacaftor–tezacaftor–ivacaftor plus ivacaftor for eligible people aged at least 12 yearsThe current Kaftrio tablet SmPC uses two 75 mg/50 mg/100 mg combination tablets in the morning and one ivacaftor 150 mg tablet in the evening, about 12 hours apart with fat-containing food.Specialist initiation only. Check ALT, AST and bilirubin before treatment, every three months during year one and annually thereafter, more often with liver disease. Review strong or moderate CYP3A modifiers, grapefruit and Seville orange, hepatic impairment, rash, pregnancy and missed-dose instructions.
Pancreatic enzyme replacement therapyDose lipase units individually with every fat-containing meal, snack and supplement using the CF dietitian's plan; adjust to weight, stool and dietary response and remain within the current product and CF-centre safety ceiling.Swallow enteric-coated microspheres as instructed and do not crush them. Persistent symptoms require review of timing, adherence, acid suppression, diet, constipation and alternate diagnoses; very high cumulative doses are associated with fibrosing colonopathy.