kaftrio(Trikafta)的功效与作用及副作用？

is a drug used to treat cystic fibrosis (CF) in patients with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or a responsive mutation in the CFTR gene based on in vitro data, that can help CF patients transform from a progressive disease to a more manageable chronic disease.

Effects of kaftrio (Trikafta)

The English name of kaftrio (Trikafta) is Ivacaftor, Tezacaftor, and Elexacaftor, and the Chinese name is Ivacaftor/Tezacaftor/Elexacaftor composite film sheet. Elexacaftor and tezacaftor bind to different sites on the CFTR protein and have an additive effect in promoting the cellular processing and transport of selected mutant forms of CFTR. Compared with any single molecule, it increases the amount of CFTR protein delivered to the cell surface.

The combined action of elexacaftor, tezacaftor, and ivacaftor increases the amount and function of CFTR on the cell surface, resulting in increased CFTR activity as measured by CFTR-mediated chloride transport.

kaftrio(Trikafta)

In a study evaluating 107 CF patients 12 years and older, the mean ppFEV1 at baseline was 60.9% after a 4-week open-label run-in period with kaftrio (Trikafta). The primary endpoint was the mean absolute change from baseline in ppFEV1 at week 4 of the double-blind treatment period. Key secondary efficacy endpoints were absolute changes from baseline in sweat chloride and CFQ-R respiratory domain scores at week 4.

Treatment with Trikafta resulted in a statistically significant 10% improvement in ppFEV1 compared with tezacaftor/ivacaftor. Mean improvement in ppFEV1 was observed at the first assessment on day 15.

Side effects of kaftrio (Trikafta)

The most common medications (≥5% of patients and frequency ≥1% above placebo) were headache, upper respiratory tract infection, abdominal pain, diarrhea, rash, increased alanine aminotransferase, nasal congestion, increased blood creatine phosphokinase, increased aspartate aminotransferase, rhinorrhea, rhinitis, influenza, sinusitis, and increased blood bilirubin.

Serious adverse reactions mainly include cataracts, allergic reactions, etc.

How to deal with side effects of kaftrio (Trikafta)

1. Headache: Pay attention to rest, avoid fatigue, and maintain emotional stability to avoid aggravating headache symptoms due to emotional excitement. You can also massage your head appropriately to promote local blood circulation and relieve headaches.

2. Abdominal pain: You can promote local blood circulation and improve abdominal pain through methods such as hot compresses and keeping warm. You should also quit smoking and drinking, avoid eating hard, cold, and spicy food, chew slowly when eating, and avoid overeating.

3. Diarrhea: Under the guidance of a doctor, anti-diarrheal drugs such as montmorillonite powder, medicinal charcoal, and loperamide can be used to treat diarrhea. Eat more light, easily digestible food, drink more hot water, and keep your abdomen warm at the same time.

4. Skin rash: Pay attention to skin cleanliness and hygiene every day. You can clean the skin with warm water to keep the skin moist and avoid drying the skin. When going out, you should apply sunscreen and wear a straw hat to avoid exposing the skin to the sun and avoid scratching the affected area.

5. Transaminase elevation: If ALT or AST > 5 times the upper limit of normal (ULN), or ALT or AST > 3 times ULN, and bilirubin > 2 times ULN, administration should be interrupted in time and laboratory tests should be conducted closely until the abnormality disappears.

6. Anaphylaxis: If signs or symptoms of severe allergic reaction occur during treatment, kaftrio (Trikafta) needs to be discontinued and appropriate treatment should be carried out.

7. Cataracts: It is recommended that pediatric patients starting kaftrio (Trikafta) treatment have baseline and follow-up eye examinations.

If intolerable side effects occur, you should consult a doctor promptly for professional treatment.

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