(296) Positive longitudinal effects of elexacaftor/tezacaftor/ivacaftor across multiple domains of physical and mental health: Pre-post findings in an Italian sample

Background: Italy recently initiated elexacaftor/tezacaftor/ivacaftor for adolescents and adults with CF. Although clinical trial results have shown positive effects on lung function and BMI, little data is available on elexacaftor/tezacaftor/ivacaftor’s effects on gastrointenstinal (GI) symptoms, cognitive processing, mental health and health-related quality of life (HRQoL). This investigation evaluated the global effects of this modulator in a short-term longitudinal study, assessing patients prior to starting the new modulator and 3 months later.

Methods: Sixty consecutive people with CF (pwCF) (F/M= 29/31) who were pancreatic insufficient, ages 12-58 years (M age= 26.8; SD= 9.1) participated. Baseline measures, given within 2 weeks of initiating this new modulator included: GI Symptom Tracker [1][2] (4 subscales: Abdominal Symptoms, Stools, Eating Challenges, Adherence Challenges {to enzymes}, Symbol Digit Modality Test (SDMT), PHQ-9 {depression}, GAD-7 {anxiety}, and CFQ-R. These measures were administered again 3 months later with no attrition over time.

Results: This was a within‐subject, cohort design using each participant as his/her own control. Statistically significant improvements were found from pre to 3 months post-initiation of elexacaftor/tezacaftor/ivacaftor on a majority of measures. As shown previously, both lung function and BMI increased. In addition, improvements were found in their ability to eat the required amount of calories and take enzymes as prescribed, cognitive processing, reductions in depressive symptoms, and better HRQoL in several domains (see Table 1). No significant changes were observed in Abdominal Symptoms, Stools, and anxiety.

Conclusions: This is the first study to evaluate the effects of elexacaftor/tezacaftor/ivacaftor on the whole person. Significant improvements were found across a variety of domains, including GI symptoms, health outcomes, cognitive processing, mental health and HRQoL. Additional longitudinal data are being collected at 6 months and 1 year.

Acknowledgements:

References: [1] Quittner AL, Alpern A N, Wolin D, McLeod L, Downey C, Nelson L, & Khandelwal N (2017). Psychometric analyses of a new gi symptom tracker for cystic fibrosis. Pediatric Pulmonology, 52, S453.

[2] Graziano S, Alghisi F, Ciciriello F, Lucidi V, Tabarini P, Quittner AL (2019). Italian Translation of Gastrointestinal (GI) Symptom Tracker in Patients with Cystic Fibrosis. Journal of Cystic Fibrosis,18, S175-S176.