Background: Italy initiated Elexacaftor/Tezacaftor/Ivacaftor (ETI) for people with cystic fibrosis (pwCF) in July 2021. It has been led to dramatic improvements in lung function, BMI, sweat chloride, and respiratory symptoms. However, little data is available on side-effects or effects on a broad range of outcomes. Research question: How does ETI affect mental health, cognitive processing, neuropsychological side-effects, GI symptoms, and HRQoL over time? Study design: AND METHODS This was a prospective, "real world" longitudinal study. Participants were recruited consecutively and evaluated at initiation T0 and after 1-month (T1), 3-months (T2), and 6-months (T3). Assessments included depression (PHQ-9), anxiety (GAD-7), cognition (SDMT), GI Symptom Tracker, and HRQoL (CFQ-R). Based on literature, an ad-hoc questionnaire was developed to assess side-effects: insomnia, headache, memory problems, "brain fog" and concentration problems. Following descriptive analyses, longitudinal data were analyzed using mixed models for repeated measures (MMRM), controlling for age and gender when appropriate. Results: Ninety-two consecutive pwCF (F/M=46/46; age M=25.4 years) participated. FEV1 increased initially then remained stable. BMI also increased significantly from T0 to T3 (p<0.01). Depression improved from T0 to T1 (p<0.001), however no changes in anxiety were found. Cognitive processing improved from T0 to subsequent assessments. Positive changes were reported on the GI Symptom Tracker for Stools and Adherence Challenges, however, no changes were found for Abdominal Pain and Digestion. Side-effects occurred in 10%-29%, with no reduction over time; insomnia increased significantly across time. Women reported more side-effects than men (i.e., insomnia, headache, concentration problems, brain fog). Interpretation: This prospective study evaluated the effects of ETI using multiple measures. Significant improvements were found in many domains, however, side-effects were reported by a substantial proportion of pwCF, with no improvements over time. Women reported more side-effects than men. PwCF should be followed systematically to assess the frequency of side-effects after starting this new modulator.
Longitudinal effects of Elexacaftor/Tezacaftor/Ivacaftor: Multi-dimensional assessment of neuropsychological ide-effects, physical and mental health outcomes in adolescents and adults / Graziano, Sonia; Boldrini, Francesca; Pellicano, Gaia Romana; Milo, Francesco; Majo, Fabio; Cristiani, Luca; Montemitro, Enza; Alghisi, Federico; Bella, Sergio; Cutrera, Renato; Fiocchi, Alessandro Giovanni; Quittner, Alexandra; Tabarini, Paola. - In: CHEST. - ISSN 0012-3692. - (2023). [10.1016/j.chest.2023.10.043]
Longitudinal effects of Elexacaftor/Tezacaftor/Ivacaftor: Multi-dimensional assessment of neuropsychological ide-effects, physical and mental health outcomes in adolescents and adults
Graziano, Sonia;Boldrini, Francesca;Pellicano, Gaia Romana;Milo, Francesco;Cristiani, Luca;Montemitro, Enza;
2023
Abstract
Background: Italy initiated Elexacaftor/Tezacaftor/Ivacaftor (ETI) for people with cystic fibrosis (pwCF) in July 2021. It has been led to dramatic improvements in lung function, BMI, sweat chloride, and respiratory symptoms. However, little data is available on side-effects or effects on a broad range of outcomes. Research question: How does ETI affect mental health, cognitive processing, neuropsychological side-effects, GI symptoms, and HRQoL over time? Study design: AND METHODS This was a prospective, "real world" longitudinal study. Participants were recruited consecutively and evaluated at initiation T0 and after 1-month (T1), 3-months (T2), and 6-months (T3). Assessments included depression (PHQ-9), anxiety (GAD-7), cognition (SDMT), GI Symptom Tracker, and HRQoL (CFQ-R). Based on literature, an ad-hoc questionnaire was developed to assess side-effects: insomnia, headache, memory problems, "brain fog" and concentration problems. Following descriptive analyses, longitudinal data were analyzed using mixed models for repeated measures (MMRM), controlling for age and gender when appropriate. Results: Ninety-two consecutive pwCF (F/M=46/46; age M=25.4 years) participated. FEV1 increased initially then remained stable. BMI also increased significantly from T0 to T3 (p<0.01). Depression improved from T0 to T1 (p<0.001), however no changes in anxiety were found. Cognitive processing improved from T0 to subsequent assessments. Positive changes were reported on the GI Symptom Tracker for Stools and Adherence Challenges, however, no changes were found for Abdominal Pain and Digestion. Side-effects occurred in 10%-29%, with no reduction over time; insomnia increased significantly across time. Women reported more side-effects than men (i.e., insomnia, headache, concentration problems, brain fog). Interpretation: This prospective study evaluated the effects of ETI using multiple measures. Significant improvements were found in many domains, however, side-effects were reported by a substantial proportion of pwCF, with no improvements over time. Women reported more side-effects than men. PwCF should be followed systematically to assess the frequency of side-effects after starting this new modulator.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
