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P22 Medication burden in children and young people with cystic fibrosis on Elexacaftor/Tezacaftor/Ivacaftor

bmjpo · 2026-04-09 · canonical JSON source

14 visible annotations · policy: published · automated confidence ≥ 75.00%

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Aim Evaluate the impact of Elexacaftor/Tezacaftor/Ivacaftor (ETI) on reducing long-term medication burden in children and young people (CYP) with Cystic Fibrosis (CF) at our centre.Methods We conducted a retrospective review of CYP aged 4–18 years who commenced ETI between 2020-2024. The primary outcome was the number and type of long-term medications discontinued after ETI initiation. Medication classes included nebulised anti-Pseudomonas aeruginosa (PsA) therapies (Colomycin®, Bramitob®, Promixin®), mucolytics (Dornase alfa), sodium chloride supplements, fat-soluble vitamins, pancreatic enzyme replacement (Creon®), and anti-staphylococcal antibiotic prophylaxis (flucloxacillin or co-amoxiclav). Deprescribing success was defined as sustained discontinuation, supported by symptom stability and objective monitoring, including annual sweat chloride (SwCl) and serum vitamin levels,faecal elastase (FE), routine microbiology surveillance, and spirometry.Results Of 58 CYP initiated on ETI, 39 (67%) discontinued at least one long-term medication. A total of 78 medications were deprescribed, with 4 (5%) being restarted. Among the 39 patients, 14 (36%) stopped one medication, 10 (26%) stopped two, 15 (38%) stopped three. Four of these CYP restarted a single medication.Of the 78 medications stopped, 14 (18%) were nebulised anti-PsA therapies, in CYP with chronic colonisation. Patients did not re-isolate PsA during the data collection period following the cessation of nebulised anti-PsA therapy. Dornase alfa was discontinued in 11 cases (14%); one restarted due to mucous plugging observed on bronchoscopy.Sodium chloride supplements made up 26 (33%) of the stopped medications, deprescribed if SwCl levels were <30 mmol/L. Three CYP (4%) restarted due to symptom recurrence or SwCl >30 mmol/L. SwCl testing was repeated in 7 CYP. At data cut-off, 7 had not reached 12 months post-cessation, and 12 had no repeat test documented.Fat-soluble vitamins comprised 16 (20%) of the stopped medications. Five CYP maintained normal serum levels, 4 developed vitamin D deficiency requiring colecalciferol, 1 had incomplete results requiring follow up, and 6 had not reached 12 months post-cessation therefore not repeated.Creon® was stopped in 7 cases (8%), with all these CYP remaining off treatment. Faecal elastase (FE) levels sustained with 4 >200 µg/g, 1 with 179 µg/g and 2 not yet rechecked.Prophylactic antibiotics accounted for 4 (5%) medication discontinuations. These were discontinued in stable patients with no recent Staphylococcus aureus growth (<18 months). All CYP remained off prophylaxis. Two had one more antibiotic course in the 12 months post-discontinuation, one fewer, and one had not completed one year off prophylaxis.Conclusion This review demonstrates that two-thirds of our local cohort of CYP with CF experienced a reduction in long-term medication burden following ETI initiation. Most remained off discontinued treatments with minimal need for reintroduction. While monitoring varied, it generally supported safe deprescribing. ETI enabled rationalisation of long-term therapy, potentially improving quality of life. Standardised monitoring and ongoing follow-up are recommended to maintain clinical stability. All patients with outstanding monitoring at the time of data collection (e.g. SwCl, vitamin levels, FE) have since been reviewed to complete post-discontinuation assessment.