Date of Award

Spring 4-8-2024

Document Type


First Advisor

Amalia Margaret

Second Advisor

David Nichols

Third Advisor

Derek Braun


Background: Prior research has observed the relationship between psychological stressors and onset of chronic diseases as well as increased severity. This begs the question of whether psychometric qualities in Health-Related Quality of Life instruments can preliminarily predict participant risk of pathology or adverse events (AE) in clinical trials. Specifically, in studies of cystic fibrosis (CF), a genetic disease that primarily affects the lungs, pancreas, and other organs due to mucus accumulation. Methods: The study design was an ancillary analysis of a clinical trial (STOP-2) examining duration of intravenous antimicrobial treatment for pulmonary exacerbations in CF (n=908). Demographic characteristics, genotype, clinical comorbidities, microbiologic history, and lung function were included as potential confounders. The exposure of interest was psychological status at baseline, and the outcome of interest was the occurrence of AEs over the study (approximately 1 month). Patient reported measures were specific Cystic Fibrosis Respiratory Symptom Diary (CFRSD) and EuroQol-5 Dimension-5 Level (EQ-5D-DL) items deemed relevant indicators of psychological status. Each psychological status measure was assessed separately versus AEs. Spearman's rho assessed potential correlation between questionnaire items and the detection of any AEs over follow-ups, as well as with AE incidence rates including multiple AEs. Poisson regression assessed whether predictors, adjusted in accordance with a casual framework, facilitated increased AE incidence. Results: 11.6% of 908 persons experienced an AE during the course of treatment (n=105). In the bivariable analyses, selected psychological items from EQ-5D-5L, which pertained to self-care, adherence to usual activities, and anxiety or depression within the current day lacked significance in both occurrence of AEs and AE incidence rates. However, items from CFRSD, which pertained to difficulty sleeping, worry about CF, feelings of crankiness, sadness and depression, and frustration within the past 24 hours were all associated with higher presence and incidence of AEs. Following multivariate adjustment, higher summary CFRSD scores were associated with 1.10 times higher risk for AE incidence (95% CI [1.06, 1.15], p<0.001). Positive culture of B. cepacia complex at enrollment was associated with increased risk (aIRR= 3.69, 95% CI [0.76, 17.93], p<0.001). In other clinical predictors, medical history of pancreatitis was associated with 3.03 times (95% CI [1.23, 7.46], p = 0.003) higher AE incidence. After adjustment, there were no significant associations between summary EQ-5D-5L scores and risk for AE incidence. Conclusion: Psychological items in CFRSD at baseline were independently associated with AEs. Ultimately, psychological status represented by CFRSD interacted with physiological and clinical factors in prediction of AEs in STOP2 and may identify individuals at greater risk of experiencing adverse events.

Included in

Diseases Commons



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