Early anti-pseudomonal acquisition in young patients with cystic fibrosis: Rationale and design of the EPIC clinical trial and observational study

被引：103

作者：

Treggiari, Miriam M. ^{[1
,2
]}

Rosenfeld, Margaret ^{[3
,4
]}

Mayer-Hamblett, Nicole ^{[2
,3
,4
]}

Retsch-Bogart, George ^{[5
]}

Gibson, Ronald L. ^{[3
,4
]}

Williams, Judy ^{[2
,3
,4
]}

Emerson, Julia ^{[3
,4
]}

Kronmal, Richard A. ^{[2
,6
]}

Ramsey, Bonnie W. ^{[2
,3
,4
]}

机构：

[1] Univ Washington, Harborview Med Ctr, Dept Anesthesiol & Pain Med, Harborview Anesthesiol Res Ctr,Sch Med, Seattle, WA 98104 USA

[2] Cyst Fibrosis Fdn, Cyst Fibrosis Therapeut Dev Network, Seattle, WA USA

[3] Univ Washington, Dept Pediat, Childrens Hosp, Seattle, WA 98195 USA

[4] Med Reg Ctr, Seattle, WA USA

[5] Univ N Carolina, Dept Pediat, Chapel Hill, NC USA

[6] Univ Washington, Dept Biostat, Sch Publ Hlth & Community Med, Seattle, WA USA

来源：

CONTEMPORARY CLINICAL TRIALS | 2009年 / 30卷 / 03期

关键词：

Antimicrobials; Inhaled tobramycin; Ciprofloxacin; Pseudomonas aeruginosa; Mucoviscidosis; Clinical trial; INHALED TOBRAMYCIN; AERUGINOSA COLONIZATION; PULMONARY-FUNCTION; RISK-FACTORS; CHILDREN; CIPROFLOXACIN; INFECTION; INFLAMMATION; STATEMENT; INFANTS;

D O I：

10.1016/j.cct.2009.01.003

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

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 CIF 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 CIF 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. (C) 2009 Elsevier Inc. All rights reserved.

引用

页码：256 / 268

页数：13