BACKGROUND: The primary cause of morbidity and mortality in patients with cystic fibrosis (CF) is progressive obstructive pulmonary disease due to chronic endobronchial infection, particularly with Pseudomonas aeruginosa (Pa). Risk factors for and clinical impact of early Pa infection in young CF patients are less well understood. PURPOSE: The present studies are designed to evaluate risk factors and outcomes associated with early Pa acquisition, and the benefits and harms of four anti-pseudomonal treatment regimens in young CF patients initiated after the first Pa positive respiratory culture. METHODS: The Early Pseudomonas Infection Control (EPIC) program consists of two studies, a randomized multicenter trial in CF patients ages 1-12 years at first isolation of Pa from a respiratory culture, and a longitudinal cohort study enrolling Pa-negative patients. Using a factorial design, trial participants are assigned for 18 months to either anti-pseudomonal treatment on a scheduled quarterly basis (cycled therapy) or based on recovery of Pa from quarterly respiratory cultures (culture-based therapy). The study drugs include inhaled tobramycin (300 mg BID) for 28 days, combined with either oral ciprofloxacin (15-20 mg/kg BID) or oral placebo for 14 days. The primary endpoints of the trial are the time to pulmonary exacerbation requiring IV antibiotics or hospitalization for respiratory symptoms, and the proportion of patients with new Pa-positive respiratory cultures during the study. The broad goals of the observational study are to describe the risk factors and outcomes associated with early acquisition of Pa. 306 patients were randomized in the clinical trial and 1787 were enrolled in the cohort study. CONCLUSIONS: These companion studies will provide valuable epidemiological and microbiological information on early CF lung disease and Pa acquisition, and safety and clinical efficacy data on anti-pseudomonal treatment strategies for early Pa infections in the airways of young children with CF.
RCT Entities:
BACKGROUND: The primary cause of morbidity and mortality in patients with cystic fibrosis (CF) is progressive obstructive pulmonary disease due to chronic endobronchial infection, particularly with Pseudomonas aeruginosa (Pa). Risk factors for and clinical impact of early Pa infection in young CFpatients are less well understood. PURPOSE: The present studies are designed to evaluate risk factors and outcomes associated with early Pa acquisition, and the benefits and harms of four anti-pseudomonal treatment regimens in young CFpatients initiated after the first Pa positive respiratory culture. METHODS: The Early PseudomonasInfection Control (EPIC) program consists of two studies, a randomized multicenter trial in CFpatients ages 1-12 years at first isolation of Pa from a respiratory culture, and a longitudinal cohort study enrolling Pa-negative patients. Using a factorial design, trial participants are assigned for 18 months to either anti-pseudomonal treatment on a scheduled quarterly basis (cycled therapy) or based on recovery of Pa from quarterly respiratory cultures (culture-based therapy). The study drugs include inhaled tobramycin (300 mg BID) for 28 days, combined with either oral ciprofloxacin (15-20 mg/kg BID) or oral placebo for 14 days. The primary endpoints of the trial are the time to pulmonary exacerbation requiring IV antibiotics or hospitalization for respiratory symptoms, and the proportion of patients with new Pa-positive respiratory cultures during the study. The broad goals of the observational study are to describe the risk factors and outcomes associated with early acquisition of Pa. 306 patients were randomized in the clinical trial and 1787 were enrolled in the cohort study. CONCLUSIONS: These companion studies will provide valuable epidemiological and microbiological information on early CF lung disease and Pa acquisition, and safety and clinical efficacy data on anti-pseudomonal treatment strategies for early Pa infections in the airways of young children with CF.
Authors: Nicole Beydon; Stephanie D Davis; Enrico Lombardi; Julian L Allen; Hubertus G M Arets; Paul Aurora; Hans Bisgaard; G Michael Davis; Francine M Ducharme; Howard Eigen; Monika Gappa; Claude Gaultier; Per M Gustafsson; Graham L Hall; Zoltán Hantos; Michael J R Healy; Marcus H Jones; Bent Klug; Karin C Lødrup Carlsen; Sheila A McKenzie; Francçois Marchal; Oscar H Mayer; Peter J F M Merkus; Mohy G Morris; Ellie Oostveen; J Jane Pillow; Paul C Seddon; Michael Silverman; Peter D Sly; Janet Stocks; Robert S Tepper; Daphna Vilozni; Nicola M Wilson Journal: Am J Respir Crit Care Med Date: 2007-06-15 Impact factor: 21.405
Authors: Ronald L Gibson; Julia Emerson; Sharon McNamara; Jane L Burns; Margaret Rosenfeld; Ann Yunker; Nicole Hamblett; Frank Accurso; Mark Dovey; Peter Hiatt; Michael W Konstan; Richard Moss; George Retsch-Bogart; Jeffrey Wagener; David Waltz; Robert Wilmott; Pamela L Zeitlin; Bonnie Ramsey Journal: Am J Respir Crit Care Med Date: 2002-12-12 Impact factor: 21.405
Authors: M Rosenfeld; R Gibson; S McNamara; J Emerson; K S McCoyd; R Shell; D Borowitz; M W Konstan; G Retsch-Bogart; R W Wilmott; J L Burns; P Vicini; A B Montgomery; B Ramsey Journal: J Pediatr Date: 2001-10 Impact factor: 4.406
Authors: M Rosenfeld; R L Gibson; S McNamara; J Emerson; J L Burns; R Castile; P Hiatt; K McCoy; C B Wilson; A Inglis; A Smith; T R Martin; B W Ramsey Journal: Pediatr Pulmonol Date: 2001-11
Authors: J L Burns; R L Gibson; S McNamara; D Yim; J Emerson; M Rosenfeld; P Hiatt; K McCoy; R Castile; A L Smith; B W Ramsey Journal: J Infect Dis Date: 2000-12-27 Impact factor: 5.226
Authors: Carolyn J Dakin; Andrew H Numa; He Wang; John R Morton; Calypso C Vertzyas; Richard L Henry Journal: Am J Respir Crit Care Med Date: 2002-04-01 Impact factor: 21.405
Authors: Nicole Mayer-Hamblett; Richard A Kronmal; Ronald L Gibson; Margaret Rosenfeld; George Retsch-Bogart; Miriam M Treggiari; Jane L Burns; Umer Khan; Bonnie W Ramsey Journal: Pediatr Pulmonol Date: 2011-08-09
Authors: Scott D Sagel; Brandie D Wagner; Assem Ziady; Tom Kelley; John P Clancy; Monica Narvaez-Rivas; Joseph Pilewski; Elizabeth Joseloff; Wei Sha; Leila Zelnick; Kenneth D R Setchell; Sonya L Heltshe; Marianne S Muhlebach Journal: J Cyst Fibros Date: 2019-12-20 Impact factor: 5.482
Authors: Zhe Hui Hoo; Elizabeth Coates; Chin Maguire; Hannah Cantrill; Nadia Shafi; Edward F Nash; Angela McGowan; Stephen J Bourke; William G Flight; Thomas V Daniels; Julia A Nightingale; Mark I Allenby; Rachael Curley; Martin J Wildman Journal: Eur J Clin Microbiol Infect Dis Date: 2018-08-18 Impact factor: 3.267
Authors: Lyndia C Brumback; Arthur Baines; Felix Ratjen; Stephanie D Davis; Stephen L Daniel; Alexandra L Quittner; Margaret Rosenfeld Journal: Pediatr Pulmonol Date: 2014-04-29
Authors: Nicole Mayer-Hamblett; Margaret Rosenfeld; Ronald L Gibson; Bonnie W Ramsey; Hemantha D Kulasekara; George Z Retsch-Bogart; Wayne Morgan; Daniel J Wolter; Christopher E Pope; Laura S Houston; Bridget R Kulasekara; Umer Khan; Jane L Burns; Samuel I Miller; Lucas R Hoffman Journal: Am J Respir Crit Care Med Date: 2014-08-01 Impact factor: 21.405
Authors: Edith T Zemanick; J Kirk Harris; Steven Conway; Michael W Konstan; Bruce Marshall; Alexandra L Quittner; George Retsch-Bogart; Lisa Saiman; Frank J Accurso Journal: J Cyst Fibros Date: 2009-10-14 Impact factor: 5.482