靶向药kaftrio(Trikafta)的中文说明书

kaftrio (Trikafta) consists of three components: Elexacaftor, Tezacaftor and Ivacaftor, which work by synergistically regulating the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The R&D company is Vertex Pharmaceuticals of the United States. In 2019, the US FDA approved its marketing, and was subsequently approved in the European Union, Canada, Australia and other countries.

Indications

kaftrio (Trikafta) is a combination preparation composed of CFTR potentiators Elexacaftor, Tezacaftor‌ and ‌Ivacaftor‌. It is suitable for the treatment of cystic fibrosis (CF) patients aged 2 years and above who carry at least one F508del mutation in the CFTR gene or a CFTR gene mutation confirmed to be responsive to the drug based on clinical and/or in vitro data.

If the patient's genotype is unknown, an FDA-approved cystic fibrosis mutation test should be used to confirm the presence of at least one mutation relevant to the above indications.

Mechanism of action

The three active ingredients of kaftrio (Trikafta) improve the function of CFTR protein through synergistic effect:

‌Elexacaftor and Tezacaftor‌: Promote the transport of CFTR protein to the cell surface and increase the amount of functional protein.

‌Ivacaftor‌: Prolongs the opening time of CFTR protein on the cell surface, enhances chloride channel activity, thereby diluting mucus.

Common adverse reactions

Including headache, flu-like symptoms (fever, chills, body aches), cold symptoms (nasal congestion, sinus pain, sore throat), diarrhea, abdominal pain, rash, and abnormal laboratory indicators (such as elevated bilirubin, creatine phosphokinase, and liver enzymes).

Recommended articles:

Serious risks and warnings

‌Liver damage and liver failure‌

It may cause severe liver damage or even liver failure (including transplantation and fatal cases).

‌Monitoring requirements‌

Regularly test liver function before and after medication; high-risk patients need to increase the frequency of testing.

‌Indications for discontinuation‌

Right upper quadrant pain, nausea and vomiting, dark urine, jaundice, loss of appetite or confusion.

Allergic reaction‌

May cause angioedema or anaphylactic shock. It is contraindicated for people with known allergies.

‌Contraindicated groups‌

Patients with advanced liver disease (such as cirrhosis, hepatic encephalopathy) should use it with caution, and the benefit-risk ratio needs to be evaluated.

‌Risk of cataracts in children‌

Drugs containing ivacaftor may induce cataracts in children. Regular eye examinations are required before use and during treatment.

‌Contraindicated groups‌

The safety of children under 2 years old is unknown.

Usage and Dosage

Route of administration: Oral administration only.

‌Tablet‌: Swallow the tablet whole (do not chew/crush).

‌Those who have difficulty swallowing‌: Consult your doctor to switch to granules.

‌Dosage frequency‌: 2 times a day (morning + evening, about 12 hours apart).

Must be taken with fatty foods: Applicable foods include butter, vegetable oil, eggs, peanut butter, nuts, meat, and full-fat dairy products (whole milk/cheese/yoghurt).

Recommended articles:

Main ingredients

Active ingredients: elexacaftor, tezacaftor and ivacaftor.

Inactive ingredients: hypromellose, succinate acetate, magnesium stearate, etc.

Contraindications during medication‌

‌Activity restrictions‌

Avoid driving or high-risk operations after taking medication until it is confirmed that the medication does not affect reaction ability.

‌Dietary taboos‌

‌Strictly avoid‌grapefruit and its products (may cause serious adverse reactions).

‌Medication Management‌

Before adding new prescription drugs/health products, you must consult a doctor or pharmacist.

Drug overdose

Unclear.

Drug interactions

‌ Drugs with high risk of interactions when shared with kaftrio (Trikafta):

‌Antibiotics‌

Rifampin, rifabutin, telithromycin, clarithromycin, erythromycin.

‌Anti-epileptic drugs‌

phenobarbital (phenobarbital), carbamazepine (carbamazepine), phenytoin (phenytoin).

‌Herbal preparations‌

St. John’s wort.

‌Antifungals‌

Ketoconazole, itraconazole, posaconazole, voriconazole, fluconazole.

Note: Due to limited space, please refer to the original drug instructions for details. Please follow your doctor's instructions for specific medication.

Storage method

Store kaftrio (Trikafta) at room temperature between 20°C-25°C. Please keep out of reach of children.