TY - JOUR
T1 - Positive and negative impacts of elexacaftor/tezacaftor/ivacaftor
T2 - Healthcare providers' observations across US centers
AU - Bathgate, Christina J.
AU - Muther, Emily
AU - Georgiopoulos, Anna M.
AU - Smith, Beth
AU - Tillman, Laura
AU - Graziano, Sonia
AU - Verkleij, Marieke
AU - Lomas, Paula
AU - Quittner, Alexandra
N1 - Funding Information: Christina J. Bathgate reports grants, funding, personal fees, and travel reimbursement from the Cystic Fibrosis Foundation, and personal fees from Vertex Pharmaceuticals. Emily Muther reports grants, funding, honoraria, and travel reimbursement from the Cystic Fibrosis Foundation. Anna M. Georgiopoulos reports personal fees from the Belgian Cystic Fibrosis Foundation/King Baudouin Foundation; grants, personal fees, and travel reimbursement from Cystic Fibrosis Foundation; grants from the Dutch Cystic Fibrosis Foundation; travel reimbursement from the European Cystic Fibrosis Society; travel reimbursement from the French Cystic Fibrosis Society; personal fees from the Italian Cystic Fibrosis Research Foundation; personal fees from Johns Hopkins University/DKBmed; grants from the National Heart, Lung, and Blood Institute; personal fees from Saudi Pediatric Pulmonology Association; grants and personal fees from Vertex Pharmaceuticals; and personal fees from Virginia Commonwealth University. Beth Smith reports grants, personal fees, and travel reimbursement from the Cystic Fibrosis Foundation and grants from the New York State Office of Mental Health, and personal fees from Vertex Pharmaceuticals. Sonia Graziano reports personal fees from Vertex Pharmaceuticals. Marieke Verkleij reports grants from the Dutch Cystic Fibrosis Foundation, speaker and travel reimbursement from the European Cystic Fibrosis Society, and grants from Vertex Pharmaceuticals. Paula Lomas reports employment at the Cystic Fibrosis Foundation. Alexandra Quittner reports grants from the NIH, Cystic Fibrosis Foundation, and the American Cochlear Implant Alliance, and consulting fees from Vertex Pharmaceuticals, Insmed Inc., and AN2 Inc. Laura Tillman declares no conflict of interest. Publisher Copyright: © 2023 Wiley Periodicals LLC.
PY - 2023/9
Y1 - 2023/9
N2 - Background: Elexacaftor/tezacaftor/ivacaftor (ETI) has been associated with unprecedented clinical improvements, transforming the management of cystic fibrosis (CF). However, side effects with implications for safety and well-being have been reported, including neuropsychiatric changes. This study aimed to better characterize the emerging positive and negative impacts of ETI. Methods: The Cystic Fibrosis Foundation's Mental Health Advisory Committee distributed a 26-item survey to US CF care teams to assess clinician observations of patient-reported experiences with ETI. Survey responses measured the prevalence of these effects in five domains: (1) positive physical and psychological effects, (2) sleep difficulties, (3) cognitive difficulties, (4) worsening mental health, and (5) concerns about the future and finances. Results: Seventy-five healthcare providers responded from a pediatric, adult, and combined centers. Positive physical effects of ETI and increased optimism were reported in the upper quartiles (50%–100%) and rated as having a significant impact on daily functioning. Sleep and cognitive difficulties were reported in 1%–24%, with slight impacts on functioning, and psychological symptoms (e.g., increased stress, depression, anxiety) and new psychiatric medications were reported in 1%–24%, with moderate impacts. Concerns about the future were reported in 1%–24%, with minimal impacts. Conclusion: Across US centers, providers most often observed positive physical effects of ETI. However, a variety of negative side effects were also reported, including sleep disruptions and worsening psychological functioning, which should be systematically monitored by CF teams. These national-level data are a first step in evaluating the prevalence and consequences of these side effects and can directly inform future studies.
AB - Background: Elexacaftor/tezacaftor/ivacaftor (ETI) has been associated with unprecedented clinical improvements, transforming the management of cystic fibrosis (CF). However, side effects with implications for safety and well-being have been reported, including neuropsychiatric changes. This study aimed to better characterize the emerging positive and negative impacts of ETI. Methods: The Cystic Fibrosis Foundation's Mental Health Advisory Committee distributed a 26-item survey to US CF care teams to assess clinician observations of patient-reported experiences with ETI. Survey responses measured the prevalence of these effects in five domains: (1) positive physical and psychological effects, (2) sleep difficulties, (3) cognitive difficulties, (4) worsening mental health, and (5) concerns about the future and finances. Results: Seventy-five healthcare providers responded from a pediatric, adult, and combined centers. Positive physical effects of ETI and increased optimism were reported in the upper quartiles (50%–100%) and rated as having a significant impact on daily functioning. Sleep and cognitive difficulties were reported in 1%–24%, with slight impacts on functioning, and psychological symptoms (e.g., increased stress, depression, anxiety) and new psychiatric medications were reported in 1%–24%, with moderate impacts. Concerns about the future were reported in 1%–24%, with minimal impacts. Conclusion: Across US centers, providers most often observed positive physical effects of ETI. However, a variety of negative side effects were also reported, including sleep disruptions and worsening psychological functioning, which should be systematically monitored by CF teams. These national-level data are a first step in evaluating the prevalence and consequences of these side effects and can directly inform future studies.
KW - Trikafta
KW - anxiety
KW - cognitive difficulties
KW - depression
KW - elexacaftor/tezacaftor/ivacaftor
KW - insomnia
KW - mental health
UR - http://www.scopus.com/inward/record.url?scp=85161422662&partnerID=8YFLogxK
U2 - https://doi.org/10.1002/ppul.26527
DO - https://doi.org/10.1002/ppul.26527
M3 - Article
C2 - 37265418
SN - 8755-6863
VL - 58
SP - 2469
EP - 2477
JO - Pediatric pulmonology
JF - Pediatric pulmonology
IS - 9
ER -