People with cystic fibrosis' experience of symptom burden while taking elexacaftor/tezacaftor/ivacaftor.

Despite improvements seen with elexacaftor/tezacaftor/ivacaftor (ETI), many people with CF (PWCF) still experience symptom burden. This study aimed to evaluate current perceived symptom burden and contrast it with PWCF's recollection of symptoms before starting ETI.Participants were 367 PWCF, ages 7-74 years (M age = 26.2), in the "Your ETI" (yETI) study. PWCF or caregivers of children (7-12 years) completed the Change in Perceptions of Health on ETI (CHETI) survey which assessed how often they were bothered by symptoms (respiratory, gastrointestinal/nutrition, sinus/pain, sleep/fatigue, and mental health/cognitive symptoms) before starting ETI and in the past month. A change score was calculated and categorized as less bothered, stable, or more bothered. Age differences were investigated.Participants were on ETI for an average of 3.9 years. A notable percentage reported being less bothered by respiratory symptoms including cough (72%) and chest tightness (66%), and gastrointestinal symptoms including stomach pain/discomfort (48%), constipation, gas, bloating (46%), and difficulty gaining weight (51%). A smaller proportion indicated more bother related to weight gain (26%), increased appetite (23%), sleep difficulties (17%), and mental health/cognitive symptoms (14-22%). Participants reported the most current bother from constipation/gas/bloating (48%), anxiety (44%), sleep difficulties (44%), and anger/irritability (43%). Adults, notably those >36 years old, typically reported these symptoms more burdensome.While PWCF were bothered less by symptoms now compared to before starting ETI, some symptoms still caused a high degree of bother, particularly among 'older' adults with CF. Findings underscore the need for ongoing strategies to monitor and manage non-respiratory symptoms.

Duke Scholars

Author Megan Von Isenburg