demirel, nadir2021-04-202021-04-202020-02https://hdl.handle.net/11299/219385University of Minnesota M.S. thesis. February 2020. Major: Epidemiology. Advisor: Joanne Billings. 1 computer file (PDF); v, 21 pages.Background: Chronic bacterial infection with thick tenacious secretions characterizes lung disease in cystic fibrosis (CF). Historically, thiol derivatives such as N-acetylcysteine (NAC), administered by inhalation of a nebulized solution, were used as mucolytics in CF, but are currently not recommended because of lack of evidence on their safety and efficacy. Previous studies have only focused on short-term effects of nebulized NAC. Objective: Our objective was to examine the rate of decline in forced expiratory volume in the first second of expiration (FEV1) in children with CF who received inhaled NAC and children who did not. Methods: The study population comprised children with CF who received care at two large, affiliated CF centers. Lung function data was compared between the treated and untreated groups using percent predicted (pp) FEV1. Results: Among 199 children younger than 6 years of age, mean pp FEV1 at age 6 did not differ between 107 children previously treated with NAC versus 92 children who did not receive NAC (P=.07). Use of dornase alfa and hypertonic saline was significantly less in the NAC group (P<.001). Among 235 children with CF aged >6 and ≤18 years, the rate of pp FEV1 decline was faster in children who did not receive NAC (slope, −1.47 vs -0.57; P<.001). Mean pp FEV1 at 18 years of age did not differ between 32 children treated with NAC versus 20 children who did not receive NAC (P=.06). No serious adverse events related to NAC treatment were reported. Conclusions: Long-term daily use of inhaled NAC is well tolerated and appears to decrease the rate of decline in pp FEV1 in children with CF. Prospective studies are needed to validate these findings.enThesis or Dissertation