| Autor: |
Sanders DB; Department of Pediatrics, School of Medicine, Indiana University, Indianapolis, Indiana., Bartz TM; Collaborative Health Studies Coordinating Center, Department of Biostatistics., Zemanick ET; Department of Pediatrics, School of Medicine, University of Colorado, Aurora, Colorado; and., Hoppe JE; Department of Pediatrics, School of Medicine, University of Colorado, Aurora, Colorado; and., Hinckley Stukovsky KD; Collaborative Health Studies Coordinating Center, Department of Biostatistics., Cogen JD; Department of Pediatrics, School of Medicine, University of Washington, Seattle, Washington., Bendy L; Department of Pediatrics, School of Medicine, Indiana University, Indianapolis, Indiana., McNamara S; Department of Pediatrics, School of Medicine, University of Washington, Seattle, Washington., Enright E; Collaborative Health Studies Coordinating Center, Department of Biostatistics., Kime NA; Collaborative Health Studies Coordinating Center, Department of Biostatistics., Kronmal RA; Collaborative Health Studies Coordinating Center, Department of Biostatistics., Edwards TC; Department of Health Systems and Population Health and., Morgan WJ; Department of Pediatrics, College of Medicine, University of Arizona, Tucson, Arizona., Rosenfeld M; Department of Pediatrics, School of Medicine, University of Washington, Seattle, Washington. |
| Abstrakt: |
Rationale: Despite the high prevalence and clear morbidity of cystic fibrosis (CF) pulmonary exacerbations (PEx), there have been no published clinical trials of outpatient exacerbation management. Objectives: To assess the feasibility of a pediatric clinical trial in which treatment of mild PEx is assigned randomly to immediate oral antibiotics or tailored therapy (increased airway clearance alone with oral antibiotics added only for prespecified criteria). The outcome on which sample size was based was the proportion of tailored therapy participants who avoided oral antibiotics during the 28 days after randomization. Methods: In this randomized, open-label, pilot feasibility study at 10 U.S. sites, children 6-18 years of age with CF were enrolled at their well baseline visits and followed through their first randomized PEx. Results: One hundred twenty-one participants were enrolled, of whom 94 (78%) reported symptoms of PEx at least once; of these, 81 (86%) had at least one exacerbation that met randomization criteria, of whom 63 (78%) were randomized. Feasibility goals were met, including enrollment, early detection of symptoms of PEx, and ability to randomize. Among the 33 participants assigned to tailored therapy, 10 (30%) received oral antibiotics, while 29 of 30 (97%) assigned to immediate antibiotics received oral antibiotics. The avoidance of oral antibiotics in 70% (95% confidence interval, 54-85%) was statistically significantly different from our null hypothesis that <10% of participants assigned to the tailored therapy arm would avoid antibiotics. Conclusions: Our pilot study demonstrates that conducting a randomized trial of oral antibiotic treatment strategies for mild PEx in children with CF is feasible and that assignment to a tailored therapy arm may reduce antibiotic exposure. Clinical trial registered with www.clinicaltrials.gov (NCT04608019). |
