CF – CFTR modulators — RACP Paediatrics MCQ

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HardRespiratoryCF – CFTR modulatorsRACP Paediatrics

A 5-year-old with CF has the G551D mutation (class III gating mutation). What targeted therapy is indicated for this specific mutation?

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Correct answer: C — Ivacaftor

Ivacaftor is a CFTR potentiator that is specifically indicated for gating mutations including G551D. It significantly improves lung function, weight, and sweat chloride levels. Lumacaftor/ivacaftor is used for the more common F508del homozygous genotype. Elexacaftor/tezacaftor/ivacaftor (Trikafta) is now available for patients with at least one F508del allele.

Reference: RACP Paediatric Curriculum – Respiratory; RCH Melbourne – 2023 – CF CPG