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خرید پکیج
تعداد آیتم قابل مشاهده باقیمانده : -37 مورد

Selection of CFTR modulator therapy for patients with cystic fibrosis who are ≥2 years

Selection of CFTR modulator therapy for patients with cystic fibrosis who are ≥2 years

CF: cystic fibrosis; CFTR: cystic fibrosis transmembrane regulator; ETI: elexacaftor-tezacaftor-ivacaftor (TRIKAFTA or KAFTRIO); VTD: vanzacaftor-tezacaftor-deutivacaftor (ALYFTREK).

* CFTR mutations eligible for ETI and VTD include F508del (regardless of the mutation at the other allele) and more than 270 less common mutations. An additional 31 mutations (all of which are rare) are approved for VTD but not for ETI. For specific mutations, refer to UpToDate content on CFTR modulators or to the manufacturer's prescribing information[1,2].

¶ Benefits and adverse effects of VTD and ETI are generally similar and include improvements in pulmonary function, nutrition, and quality of life and reduced frequency of exacerbations. VTD has the advantage of once-daily dosing and causes a greater reduction in sweat chloride. ETI may be less costly, and more data are available on medium-term outcomes including durability of benefits across the first few years of treatment.

Δ For patients who are not eligible for any available CFTR modulator because they have a rare CFTR genotype, consider theratyping or theranostics to determine if either of their mutations are responsive to one of the available drugs; this in vitro testing is available at several specialized CF centers. If so, they may be able to seek individual approval for insurance coverage. Other options might include a medication trial, ideally through enrollment in a clinical trial.

References:
  1. ALYFTREK (vanzacaftor, tezacaftor, and deutivacaftor) tablets. Vertex Pharmaceuticals Incorporated. Revised December 2024. https://pi.vrtx.com/files/uspi_vanzacaftor_tezacaftor_deutivacaftor.pdf (Accessed on January 14, 2025).
  2. TRIKAFTA (elexacaftor/tezacaftor/ivacaftor; ivacaftor) tablets and oral granules. Vertex Pharmaceuticals Incorporated. Revised December 2024. https://pi.vrtx.com/files/uspi_elexacaftor_tezacaftor_ivacaftor.pdf (Accessed on January 14, 2025).
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