kaftrio治疗囊性纤维化效果怎么样?

Kaftrio is effective in treating cystic fibrosis and can improve the symptoms and quality of life of patients with cystic fibrosis through different mechanisms. Kaftrio is a combination of three drugs: elexacaftor, tezacaftor and ivacaftor. This medication regimen can significantly improve lung function, sweat chloride, and respiratory symptoms. Elexacaftor and tezacaftor can increase the amount of mature CFTR protein on the cell surface and improve the function of ion channels, thereby alleviating the symptoms of patients. Ivacaftor can promote CFTR protein to cross the cell membrane and improve its ability to transport salt and water. This helps hydrate and clear mucus from the respiratory tract, reducing obstruction of the respiratory tract by thick mucus, thereby improving respiratory function.

kaftrio trial results for treating cystic fibrosis

In a phase 2 study of patients with cystic fibrosis homologous to the F508del gene mutation, adding the new generation CFTR corrector elexacaftor (VX-445) to tezacaftor and ivacaftor can further improve F508del-CFTR function and clinical efficacy.

research methods

This phase 3, multicenter, randomized, double-blind, active-controlled trial (NCT03525548) of elexacaftor in combination with tezacaftor and ivacaftor was conducted at 44 sites in 4 countries. Eligible participants are patients with homologous cystic fibrosis F508del mutation, age 12 years or older, stable condition, and predicted forced expiratory volume in 1 second (ppFEV1) percentage between 40% and 90% (inclusive).

After a 4-week tezacaftor plus ivacaftor run-in period, participants were randomly assigned (1:1) to 4 weeks of elexacaftor 200 mg PO once daily, plus tezacaftor 100 mg PO once daily, plus ivacaftor 150 mg PO every 12 hours for comparison.

The primary outcome was the absolute change in ppFEV1 at week 4 compared with baseline (measured at the end of the tezacaftor plus ivacaftor run-in period). Main secondary outcomes were absolute changes in perspiration and Cystic Fibrosis Questionnaire-Revised Respiratory Domain (CFQ-R RD) scores.

Research results

A total of 107 participants were randomly assigned (55 in the elexacaftor plus tezacaftor plus ivacaftor group and 52 in the tezacaftor plus ivacaftor group) and completed 4 weeks of treatment. The elexacaftor plus tezacaftor plus ivacaftor group had improvements in the primary outcome of ppFEV1 (least squares mean [LSM] treatment difference 10-0 percentage points [95% CI 7-4 to 12-6], p<0-0001) and the main secondary outcome sweat chloride concentration (LSM treatment difference -45-1 mmol/L [95% CI -50-1 to -40-1], p<0-0001), and CFQ-R RD score (LSM treatment difference 17-4 points [95% CI 11-8 to 23-0], p<0-0001) compared with the tezacaftor plus ivacaftor group. Triple therapy was well tolerated, with no discontinuations and most cases being mild or moderate.

Test conclusion

Compared with tezacaftor plus ivacaftor alone, Elexacaftor plus tezacaftor plus ivacaftor (kaftrio) can bring significant clinical benefits and has a good safety profile, showing the potential to bring transformative improvements to the lives of cystic fibrosis patients with F508del gene mutations.

price of kaftrio

As of October 11, 2023, it has not been launched in mainland China. It is not available in hospital pharmacies in mainland China, and there is no price announcement. At present, we have learned from domestic professional overseas medical service institutions that the price of kaftrio specifications 75 mg / 50 mg / 100 mg (56 tabs.) is about $123,600 per box.

Patients in need can purchase it in areas where it is already on the market, but they should go to regular hospital pharmacies to avoid being deceived. It can also be obtained through domestic professional overseas medical service organizations (such as Medical Companion Travel). The medicine can be mailed to your home. It is guaranteed to be genuine and affordable, which can reduce a lot of financial burden. However, the price is subject to various factors and is not fixed. It is recommended to consult the customer service staff for specific costs and acquisition procedures. Obtaining the medicine is guaranteed.

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References

Heijerman HGM, McKone EF, Downey DG, Van Braeckel E, Rowe SM, Tullis E, Mall MA, Welter JJ, Ramsey BW, McKee CM, Marigowda G, Moskowitz SM, Waltz D, Sosnay PR, Simard C, Ahluwalia N, Xuan F, Zhang Y, Taylor-Cousar JL, McCoy KS; VX17-445-103 Trial Group. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomized, phase 3 trial. Lancet. 2019 Nov 23;394(10212):1940-1948. doi: 10.1016/S0140-6736(19)32597-8. Epub 2019 Oct 31. Erratum in: Lancet. 2020 May 30;395(10238):1694. PMID: 31679946; PMCID: PMC7571408.