Author ORCID Identifier
Date of Award
Master of Science (MS)
Shi Huh Samuel Shan
Laryssa D. Frederick
Background: Cystic fibrosis (CF) is a severe genetic disorder that primarily impacts the digestive and pulmonary systems. The most frequent Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) mutation is F508del which accounts for 90% of CF patients. Cystic fibrosis patients undergo multiple treatments daily to reduce the physical and psychological adverse CF. Trikafta combines three major chemical compounds: tezacaftor, ivacaftor, and elexacaftor. The drug assists the defected proteins to function normally. Trikafta is intended for CF patients 12 years or older with at least one gene of F508del, but the study only targets 18 years or older. This study evaluates the difference in health-related quality of life (HRQOL) between CF patients taking Trikafta compared to CF patients not taking Trikafta and the length of time on Trikafta. The study also evaluates the differences in depression and anxiety levels between CF patients taking Trikafta compared to CF patients not taking Trikafta.
Methods: A cross-sectional survey containing Cystic Fibrosis Questionnaire-Revised (CFQ-R), General Anxiety Disorder-7 (GAD-7), and Patient Health Questionnaire (PHQ)-9 was utilized to be administered by CF patients from closed and certified social media groups. CFQ-R is used to evaluate the Health-Related-Quality-of-Life (HRQOL) in CF patients, where GAD-7 and PHQ-9 measure levels of anxiety and depression, respectively. The survey divides the participants into two groups; patients who are not administering Trikafta versus patients who are administering Trikafta.
Results: 59 adults with CF participated (3 were not eligible and were excluded) aged 18 to 47 (M=25.6 yr.), 37.5% are males, and 62.5% are females. The majority were Caucasian (71.4%, n=40). Approximately 57.1% of patients reported taking Trikafta, and 42.9% reported not taking Trikafta. 73.2% reported having the F508del CFTR gene mutation. Trikafta users (M=2.9, SD=0.5) compared non-Trikafta users (M=2.2, SD=0.7) revealed significantly better average CFQ-R scores, t(51)=4.2, (pp=0.006). For depression, Trikafta users (M=8.2, SD=7.6) compared to non-Trikafta users (M=12.5, SD=6.1) indicating significantly lower PHQ-9 scores, t(51) = -2.2, (p=0.017). The association between length of time taking Trikafta and overall quality of life was not statistically significant (p=0.90). GAD-7 (MD= -4.4, p=0.006) and PHQ-9 (MD= -4.3, p=0.017) scores from all participants indicate that Trikafta users have lower levels of anxiety and depression than non-Trikafta users.
Conclusion: The use of Trikafta for the Treatment of cystic fibrosis patients with F508del CFTR mutation positively impacts CF patients' quality of life when compared to patients who are not taking Trikafta. The association between the length of time on Trikafta and the quality-of-life score reveals a non-significant value, as further research with a sizable sample initiating the use of Trikafta could reveal more significant findings. Findings suggest that participants administering Trikafta have lower levels of anxiety and depression than those who do not.
Alrwaili, Yzeed, "Evaluating the Impact of Trikafta on the Quality of Life for Cystic Fibrosis Patients." Thesis, Georgia State University, 2022.
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