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CFTR Modulator Therapy — RACP Paediatrics MCQ

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HardRespiratoryCFTR Modulator TherapyRACP Paediatrics

A 12-year-old with cystic fibrosis has an annual lung function review. His FEV1 has declined from 85% to 72% predicted over 12 months despite adherence to treatment. He is heterozygous for F508del/G551D mutations. What targeted therapy may be beneficial?

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Correct answer: C — Ivacaftor (Kalydeco)

Ivacaftor (a CFTR potentiator) is specifically indicated for patients with at least one G551D gating mutation. It improves chloride channel function, resulting in improved FEV1, weight gain, and reduced pulmonary exacerbations. Elexacaftor/tezacaftor/ivacaftor (Trikafta) is indicated for patients with at least one F508del mutation.

Reference: Australian Cystic Fibrosis Guidelines – 2024 – CFTR Modulator Therapy