(292) Effects of Triple Combination Therapy on Quality of Life and Psychosocial Outcomes: A Longitudinal Study

Background: CFTR modulator triple combination therapy (TCT) is now available to approximately 85% of the US CF population. Clinical trials of TCT demonstrate numerous improvements in physical health and health-related quality of life (HRQoL). However, few studies have examined the effects of TCT on mental health and psychosocial outcomes, and little is known about whether gains in HRQoL are sustained over time. We aimed to describe the HRQoL and psychosocial outcomes of people with CF (pwCF) initiating TCT and explored changes in these outcomes up to one year after starting TCT.

Methods: This longitudinal study enrolled pwCF 14 years and older who were followed at a large, combined pediatric and adult CF center. Questionnaires were administered within 6 months of initiating TCT (baseline), and 3, 6, and 12 months later. Study self-report measures evaluated: HRQoL (CFQ-R), optimism, self-efficacy, medication-related beliefs (MBQ), perceived social stigma of illness, and body image. Data were also collected from medical charts on measures of health and mental health screening. Four open-ended questions were included at each timepoint to elicit qualitative data on experiences starting TCT. Longitudinal data were analyzed using linear mixed effects models for repeated measures.

Results: Sixty-three adults and adolescents with CF completed the full set of surveys at baseline. Mean participant age was 30.0 years (SD= 14.2). Fifty-four percent identified as female, 43% as male, and 2% as nonbinary. Seventy-four percent had private insurance. Mean FEV1 % predicted at baseline was 76.0 (SD= 24.1), and mean BMI was 22.9 (SD= 3.1). At 12 months, mean FEV1 % predicted was 80.8 (SD= 21.9), and mean BMI was 24.5 (SD= 4.1). On standard measures used in CF mental health screening, mean PHQ9 score in our sample at baseline was 3.4 (SD= 3.5) and mean GAD7 score was 3.4 (SD= 3.7). At 12 months, mean PHQ9 score in our sample was similar to baseline at 3.5 (SD= 4.0) and mean GAD7 score was also similar at 3.4 (SD= 3.7).

We found no statistically significant differences between the survey time points in participants’ Physical, Respiratory or Emotional functioning on the CFQ-R, but there was a significant change in Social functioning (p< 0.001). There was no statistically significant change over time in Optimism or Self-Efficacy. There was however a significant difference in CF Medication Beliefs between the four survey time points (p= 0.008 for MBQ Importance subscale), with an observed decrease in perceived importance from baseline to 12-month post.

Conclusions: Whereas lung function and BMI increased in our sample by 12 months, similar improvements were not seen in standard mental health outcomes. There also was no change over time in physical, respiratory or emotional functioning, or in optimism or self-efficacy. Only CFQ-R social functioning had changed by 12-months post, perhaps reflecting decreased COVID-related social isolation. There was also a change in medication-related beliefs, with a decrease in perceived importance of taking CF medications at 12-months post. Future directions include conducting qualitative analyses of open-ended questions and further examining data on social stigma, motivation to take medications, and body image, as well as examining relationships between outcome variables and Baseline FEV1 and BMI.

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