New Treatment Pathways for Cystic Fibrosis

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New Treatment Pathways for Cystic Fibrosis
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David Finklea, MD

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References
  1. Cystic Fibrosis Foundation. What is cystic fibrosis? https://www. cff.org/intro-cf/about-cystic-fibrosis. Accessed June 17, 2022.
  2. Middleton PG, Mall MA, Dřevínek P, et al. Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med. 2019;381(19):1809-1819. doi:10.1056/NEJMoa1908639
  3. McGarry ME, McColley SA. Cystic fibrosis patients of minority race and ethnicity less likely eligible for CFTR modulators based on CFTR genotype. Pediatr Pulmonol. 2021;56(6):1496-1503. doi:10.1002/ppul.25285
  4. O’Connor KE, Goodwin DL, NeSmith A, et al. Elexacaftor/ tezacaftor/ivacaftor resolves subfertility in females with CF: a two center case series. J Cyst Fibros. 2021;20(3):399-401. doi:10.1016/j.jcf.2020.12.011
  5. Shteinberg M, Taylor-Cousar JL, Durieu I, Cohen-Cymberknoh M. Fertility and Pregnancy in Cystic Fibrosis. Chest. 2021;160(6):2051-2060. doi:10.1016/j.chest.2021.07.024
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CHEST Physician
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Pulmonology
Author(s)
David Finklea, MD
Author(s)
David Finklea, MD

Click to view more from Pulmonology Data Trends 2022.

Click to view more from Pulmonology Data Trends 2022.

References
  1. Cystic Fibrosis Foundation. What is cystic fibrosis? https://www. cff.org/intro-cf/about-cystic-fibrosis. Accessed June 17, 2022.
  2. Middleton PG, Mall MA, Dřevínek P, et al. Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med. 2019;381(19):1809-1819. doi:10.1056/NEJMoa1908639
  3. McGarry ME, McColley SA. Cystic fibrosis patients of minority race and ethnicity less likely eligible for CFTR modulators based on CFTR genotype. Pediatr Pulmonol. 2021;56(6):1496-1503. doi:10.1002/ppul.25285
  4. O’Connor KE, Goodwin DL, NeSmith A, et al. Elexacaftor/ tezacaftor/ivacaftor resolves subfertility in females with CF: a two center case series. J Cyst Fibros. 2021;20(3):399-401. doi:10.1016/j.jcf.2020.12.011
  5. Shteinberg M, Taylor-Cousar JL, Durieu I, Cohen-Cymberknoh M. Fertility and Pregnancy in Cystic Fibrosis. Chest. 2021;160(6):2051-2060. doi:10.1016/j.chest.2021.07.024
References
  1. Cystic Fibrosis Foundation. What is cystic fibrosis? https://www. cff.org/intro-cf/about-cystic-fibrosis. Accessed June 17, 2022.
  2. Middleton PG, Mall MA, Dřevínek P, et al. Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med. 2019;381(19):1809-1819. doi:10.1056/NEJMoa1908639
  3. McGarry ME, McColley SA. Cystic fibrosis patients of minority race and ethnicity less likely eligible for CFTR modulators based on CFTR genotype. Pediatr Pulmonol. 2021;56(6):1496-1503. doi:10.1002/ppul.25285
  4. O’Connor KE, Goodwin DL, NeSmith A, et al. Elexacaftor/ tezacaftor/ivacaftor resolves subfertility in females with CF: a two center case series. J Cyst Fibros. 2021;20(3):399-401. doi:10.1016/j.jcf.2020.12.011
  5. Shteinberg M, Taylor-Cousar JL, Durieu I, Cohen-Cymberknoh M. Fertility and Pregnancy in Cystic Fibrosis. Chest. 2021;160(6):2051-2060. doi:10.1016/j.chest.2021.07.024
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New Treatment Pathways for Cystic Fibrosis
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New Treatment Pathways for Cystic Fibrosis
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Cystic fibrosis is a deadly genetic disorder, affecting 80,000 people worldwide.1,2 The disorder is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.2 This gene codes for a protein that creates epithelial channels in the respiratory track, along with other organs. Mutations in this gene can create improper ion balance, leading to thick and sticky mucus that blocks airways in the lungs and contributes to infections in people with CF (pwCF).1

Currently, there is no cure for cystic fibrosis, but newer research is looking into modulating the CFTR gene from multiple pathways by repairing, restoring, or replacing the CFTR protein.1,2 At this point, CFTR modulators are the most promising new treatments for cystic fibrosis.

CFTR modulators involve repairing the CFTR protein made from this gene. To qualify for treatment with this class of drugs, people with cystic fibrosis need to have certain CFTR mutations. Fortunately, approximately 90% of pwCF qualify for CFTR modulators.1,2 Due to this, the Cystic Fibrosis Foundation is working on finding alternative therapies that are listed below.1

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