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ABSTRACT

A review of cystic fibrosis (CF) is presented to primary care clinicians with updated information regarding current treatments as well as trends in the emerging therapies for CF. As a genetic disease, cystic fibrosis (CF) has often presented as a chronic illness in which patients have experienced a difficult journey of recurrent pulmonary exacerbations, frequent hospitalizations and eventually, premature death. In October of 2019 a major breakthrough in the management occurred with the availability of the highly effective triple, fixed-dose combination therapy of ivacaftor/tezacaftor/elexacaftor. This latest treatment is not a cure of CF, but has transformed the lives of persons with this disorder giving them a very different future. Indeed, historical management of the disease (CF has changed its course and new care guidelines are under development.) Introduction of cystic fibrosis transmembrane conductance regulator (CFTR) modulators in infancy or during the toddler years provides a hope for a distinctly healthier adulthood. The advances in cystic fibrosis gene therapy are progressing rapidly, and a breakthrough cure is moving closer. The recent incredible progress in CF research is stimulating a new hope and a promise for a longer as well as healthier life for CF patients. Keeping primary care providers up-to-date with such amazing advances in CF improves these clinicians’ engagement in CF care and helps the effort to provide excellent care for people with CF. Indeed, as detailed in this discussion, it is a new day for those with CF and clinicians caring for them.

Keywords: Cystic fibrosis, management, therapy, United States