Kalydeco的功效,购药渠道,副作用,注意事项?

Author: Medicalhalo

Release time: 2025-10-19 11:44:20

Effects of Kalydeco

Kalydeco, also known as ivacaftor, is suitable for the treatment of cystic fibrosis (CF) patients aged ≥6 years old who have a G551D mutation in the CFTR gene. It helps the CFTR gene function to better produce proteins, thereby improving the patient's lung function and other problems caused by CF, thereby alleviating the symptoms of the disease.

Clinical trials

In controlled clinical trials, the cystic fibrosis (CF) modulator drug elexacaftor/tezacaftor/ (ETI) was shown to be highly effective in patients with at least one F508del allele. Study Objectives PROMISE is a post-approval study (NCT NCT04038047) designed to understand the broad effects of ETIs after 30 months of clinical use in a more diverse U.S. patient population, with analysis planned after 6 months.

Research methods

A prospective observational study of 487 CF patients aged 12 years or older with at least one F508del allele who were first-time ETI users. Assessments were performed before ETI treatment and after 1, 3, and 6 months of treatment. Outcomes included changes in percent predicted FEV1 (ppFEV1), sweat chloride concentration, body mass index (BMI), and self-reported respiratory symptoms.

Test results

44.1% of patients entered the study using tezacaftor/Kalydeco or lumacaftor/Kalydeco, while 6.7% used ivacaftor, consistent with the F508del homolog and G551D allele, respectively. After 6 months of ETI treatment, ppFEV1 improved by 9.76 percentage points from baseline, Cystic Fibrosis Questionnaire-Revised respiratory domain score improved by 20.4 points (95% CI, 18.3 to 22.5), and sweat chloride decreased by -41.7 mmol/L (95% CI, -43.8 to -39.6). Body mass index also increased significantly.

Test conclusion

ETIs in clinical prescriptions can significantly improve lung function, respiratory symptoms and body mass index and are suitable for different groups of people who are not receiving modulator drug treatment, are using existing two-drug combination therapy, or are using Kalydeco alone. Patients in each group experienced significant reductions in sweat chloride concentrations, which correlated with improvements in ppFEV1 across the study population.

Kalydeco’s drug purchasing channels

Purchasing Kalydeco requires a legal prescription certificate, and generally needs to be obtained through the following channels:

1. Doctor’s prescription: First, you need to consult a doctor or specialist. After relevant examination and evaluation, if you are diagnosed with cystic fibrosis caused by a genetic mutation consistent with Kalydeco’s indications, and the doctor thinks that Kalydeco is beneficial to you, the doctor will issue a prescription to you.

2. Pharmacies or medical institutions: You can bring your prescription to a local legal pharmacy or medical institution to purchase Kalydeco. Please make sure that the place where you buy medicines is legally qualified to ensure the quality and safety of the medicines.

3. Online purchase: Some countries or regions allow online purchase of prescription drugs. You can purchase Kalydeco on legal pharmacy websites or electronic pharmacies. When purchasing medications online, make sure you choose a trustworthy website and follow relevant laws and regulations.

4. Patients who are inconvenient to travel far can also obtain the drugs through domestic professional overseas medical service organizations (such as Medical Companion Travel). The drugs can be mailed to their homes, which is guaranteed to be genuine and affordable. However, due to various factors, it is recommended to consult customer service personnel for specific costs and acquisition procedures.

Please note that purchasing prescription drugs requires compliance with local country or region laws and regulations, and it is best to consult a doctor, pharmacist or relevant professional for accurate guidance before purchasing.

Kalydeco side effects

Common side effects of Kalydeco include headache, upper respiratory tract infection, abdominal pain, diarrhea, rash, nasal congestion, rhinorrhea, rhinitis, influenza, sinusitis, increased alanine aminotransferase, creatine phosphokinase, aspartate aminotransferase, blood bilirubin, etc. Patients have different physiques and conditions, and the side effects after taking medication are also different.

During medication, be sure to ensure adequate rest time to avoid fatigue. When in pain, you can take painkillers under the guidance of a doctor. However, specific treatments for different side effects will vary depending on the cause and condition. It is recommended that you consult your doctor and proceed with treatment based on your doctor's diagnosis and recommendations, which may include rest, appropriate medication, adequate fluid intake, and maintaining cleanliness and hygiene. For elevations in various indicators, it is recommended that doctors conduct evaluation and further examination to understand the possible causes and formulate corresponding treatment plans.

Things to note about Kalydeco

1. For patients with a history of elevated transaminases, increased monitoring frequency of liver function tests should be considered.

2. Using Kalydeco will cause immune system diseases: immediate allergic reactions.

3. It is not recommended to co-administer Kalydeco with strong CYP3A inducers (such as rifampicin, St. John's wort)

4. It is recommended that pediatric patients starting treatment with Kalydeco undergo baseline and follow-up eye examinations.

5. People with impaired liver and kidney function must take medication under the guidance of a doctor.

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