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Adult Cystic Fibrosis

MAC & Bronchiectasis | Last Active: 5 days ago | Replies (17)

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Profile picture for tw508 @tw508

Sorry if the above post appeared to veer into "adult CF' territory. It was meant to refer to the potential benefit of using CFTR modulators (Trikafta et al) in people with BE who carry a CF mutation. It's not that unusual to carry the mutation, and most people go through life without knowing it, but it does appear to increase the risk of problems such as BE, pancreatitis, et al.

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Replies to "Sorry if the above post appeared to veer into "adult CF' territory. It was meant to..."

@tw508 I appreciate your information. Here is what I searched for. My mother also died of lung cancer at age of 77. My grandfather got TB when he served in the army. I will get into more research in CF and related mutation. I believe it will benefit most of the patients in MAC/BE forum too.

Trikafta (elexacaftor/tezacaftor/ivacaftor) is a revolutionary triple-combination medicine for Cystic Fibrosis (CF), targeting specific genetic mutations like F508del, and works as a CFTR modulator to help the protein function better, treating CF in patients 2+ years with responsive mutations, but requires liver monitoring due to risks of serious liver damage, with S895N being a specific but less common CF mutation often responsive to this therapy.
What Trikafta Does
Mechanism: It's a CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator that corrects the faulty CFTR protein, helping it move to the cell surface and function correctly, improving mucus clearance.
Components: Contains elexacaftor, tezacaftor (correctors), and ivacaftor (potentiator).

Responsiveness: Trikafta is indicated for patients with at least one F508del mutation or other mutations, that are responsive to the drug based on clinical data.
Key Considerations
Liver Issues: A major warning is potential serious liver damage or failure, requiring regular blood tests.
Dosage: Taken orally, usually twice daily, with fat-containing food.
Eligibility: For CF patients aged 2 and older with at least one F508del or another responsive mutation.