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The Journal of Heart and Lung Transplantation
International Society for Heart and Lung Transplantation.

A growing trend: CFTR modulators for cystic fibrosis lung transplant recipients

  • Don Hayes Jr.
    Correspondence
    Reprint requests: Don Hayes Jr., MD, MS, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, 3333 Burnet Ave, MLC 7041, Cincinnati, OH 45229. Telephone: (513) 803-7009. Fax: (513) 803-2183.
    Affiliations
    Division of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio

    Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, Ohio
    Search for articles by this author
  • Nirmal S. Sharma
    Affiliations
    Division of Pulmonary and Critical Care Medicine, Brigham and Women's Hospital, Boston, Massachusetts

    Department of Medicine, Harvard Medical School, Boston, Massachusetts
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Published:October 22, 2021DOI:https://doi.org/10.1016/j.healun.2021.10.003
      Life expectancy for people with cystic fibrosis (CF) has steadily improved over the past 2 decades, with a median survival of 48.4 (95% CI: 45.9-51.5) years in the United States (US) according to the 2019 US Cystic Fibrosis Foundation Patient Registry Report. This survival improvement is attributed to numerous advancements, including airway clearance technology and antimicrobial therapies as well as the standardization of clinical care guided by numerous multicenter clinical trials addressing key questions in the management of CF. More recently, the advent of cystic fibrosis transmembrane conductance regulator (CFTR) gene modulator therapy, which corrects the malfunctioning protein, started with the approval of ivacaftor in 2012 and the triple combination therapy (elexacaftor/tezacaftor/ivacaftor or Trikafta) in 2019. Trikafta greatly expanded CTFR modulator use to a larger proportion of the CF population for those people with at least one copy of the F508del mutation or one copy of 177 specified genes. These new therapies that target the malfunctioning protein have significantly altered the paradigm regarding the approach to clinical care for CF in the modern era.

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