TY - JOUR
T1 - Understanding and addressing the needs of people with cystic fibrosis in the era of CFTR modulator therapy
AU - Hisert, Katherine B
AU - Birket, Susan E
AU - Clancy, John Paul
AU - Downey, Damian G
AU - Engelhardt, John F
AU - Fajac, Isabelle
AU - Gray, Robert D
AU - Lachowicz-Scroggins, Marrah E
AU - Mayer-Hamblett, Nicole
AU - Thibodeau, Patrick
AU - Tuggle, Katherine L
AU - Wainwright, Claire E
AU - De Boeck, Kris
PY - 2023/10/3
Y1 - 2023/10/3
N2 - Cystic fibrosis is a multiorgan disease caused by impaired function of the cystic fibrosis transmembrane conductance regulator (CFTR). Since the introduction of the CFTR modulator combination elexacaftor-tezacaftor-ivacaftor (ETI), which acts directly on mutant CFTR to enhance its activity, most people with cystic fibrosis (pwCF) have seen pronounced reductions in symptoms, and studies project marked increases in life expectancy for pwCF who are eligible for ETI. However, modulator therapy has not cured cystic fibrosis and the success of CFTR modulators has resulted in immediate questions about the new state of cystic fibrosis disease and clinical challenges in the care of pwCF. In this Series paper, we summarise key questions about cystic fibrosis disease in the era of modulator therapy, highlighting state-of-the-art research and clinical practices, knowledge gaps, new challenges faced by pwCF and the potential for future health-care challenges, and the pressing need for additional therapies to treat the underlying genetic or molecular causes of cystic fibrosis.
AB - Cystic fibrosis is a multiorgan disease caused by impaired function of the cystic fibrosis transmembrane conductance regulator (CFTR). Since the introduction of the CFTR modulator combination elexacaftor-tezacaftor-ivacaftor (ETI), which acts directly on mutant CFTR to enhance its activity, most people with cystic fibrosis (pwCF) have seen pronounced reductions in symptoms, and studies project marked increases in life expectancy for pwCF who are eligible for ETI. However, modulator therapy has not cured cystic fibrosis and the success of CFTR modulators has resulted in immediate questions about the new state of cystic fibrosis disease and clinical challenges in the care of pwCF. In this Series paper, we summarise key questions about cystic fibrosis disease in the era of modulator therapy, highlighting state-of-the-art research and clinical practices, knowledge gaps, new challenges faced by pwCF and the potential for future health-care challenges, and the pressing need for additional therapies to treat the underlying genetic or molecular causes of cystic fibrosis.
U2 - 10.1016/S2213-2600(23)00324-7
DO - 10.1016/S2213-2600(23)00324-7
M3 - Article
C2 - 37699420
SN - 2213-2600
VL - 11
JO - The Lancet. Respiratory medicine
JF - The Lancet. Respiratory medicine
IS - 10
M1 - 16
ER -