Health-related quality of life, optimism and self-efficacy among people with CF beginning triple combination therapy

Background: With FDA approval in October 2019 of elexacaftor/ tezacaftor/ivacaftor, a CFTR modulator triple combination therapy (TCT), approximately 85% of the CF population was eligible to initiate this treatment Clinical trial data indicates numerous improvements in physical health and health-related quality of life (HRQoL) Real-world studies of treatment initiation recommend including mental health as an outcome meaure, not reported in clinical trials Objective: To describe the QoL and mental health outcomes of people with CF (pwCF) initiating TCT in a real-world setting Methods: This longitudinal study enrolled pwCF 14 years and older who are followed at a large, combined pediatric and adult CF center Data will be obtained at the following timepoints: within 6 months of initiating TCT (baseline), and then at 3, 6, and 12 months after baseline Study self-report measures evaluate: HRQoL (CFQ-R), optimism, perceived social stigma of illness, self-efficacy, medication-related beliefs, and body image Four open-ended questions were included to elicit qualitative data on experiences starting TCT Data from the baseline survey are reported here Results: Sixty-five adults and adolescents with CF completed the full set of surveys at baseline Mean participant age was 30 2 years (SD= 14 0) Among this group, 57% identified as female, 42% as male, and 2% as nonbinary With respect to education, 20 8% completed high school or less, 23 6% completed some college, and 45 9% completed college or above As compared to a large 2010 US sample (Quittner, et al), participants reported higher Physical functioning (t=3 0;p<0 01), lower Emotional functioning (t=-6 7;p<0 001), and lower Social functioning (t=-2 1;p<0 05) on the CFQ-R measure of HRQoL In terms of mental health, participants reported a mean score of 15 6 (SD= 5 7) on the LOT-R Optimism scale, falling in the Moderate optimism range Participants had a mean score of 32 4 (SD= 4 3) on the General Self-Efficacy Scale measure, representing a t-score of 56 (73rd percentile) Open-ended questions revealed that patients' expectations regarding initiating TCT ranged from skepticism, to cautious optimism, to high expectations for life-changing results Common hopes for TCT included reduction in treatment burden and increased quality of life, while collective fears included ineffectiveness and negative side effects Many patients identified a change in future planning in response to starting TCT, namely increased hope and ambition Conclusion: On average, pwCF in our sample who were starting TCT reported feeling moderate optimism and self-efficacy They reported better physical functioning, but worse emotional and social functioning, than a 2010 sample Whether these differences in HRQoL are due to TCT, COVID-19, or other factors requires further study Open-ended questions elicited a mixture of positive and negative feelings related to starting TCT Future analyses for this study include evaluation of key outcomes from the 3-month follow-up timepoint, including data on the mental health impact of COVID-19 Future directions include longitudinal analyses of the impact of TCT on HRQoL and mental health