Early anti-pseudomonal acquisition in young patients with cystic fibrosis: Rationale and design of the EPIC clinical trial and observational study
Early anti-pseudomonal acquisition in young patients with cystic fibrosis: Rationale and design of the EPIC clinical trial and observational study
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DOI：
10.1016/j.cct.2009.01.003
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发表时间：
2009-05-01
影响因子：
2.2
通讯作者：
Ramsey, Bonnie W.
中科院分区：
文献类型：
--
作者：
Treggiari, Miriam M.;Rosenfeld, Margaret;Ramsey, Bonnie W.
背景资料：囊性纤维化（CF）患者发病和死亡的主要原因是慢性支气管内感染（特别是铜绿假单胞菌（Pa））导致的进行性阻塞性肺疾病。年轻CIF患者早期Pa感染的风险因素和临床影响尚不清楚。目的：本研究旨在评估与早期Pa获得相关的风险因素和结局，以及在首次Pa阳性呼吸道培养后开始的年轻CF患者中四种抗假单胞菌治疗方案的益处和危害。方法：早期假单胞菌感染控制（EPIC）计划包括两项研究，一项是在1-12岁CF患者中首次从呼吸道培养中分离Pa的随机多中心试验，另一项是招募Pa阴性患者的纵向队列研究。使用析因设计。试验参与者被分配接受18个月的抗假单胞菌治疗，以预定的季度为基础（周期性治疗）或基于从季度呼吸道培养物中恢复Pa（基于培养物的治疗）。研究药物包括吸入妥布霉素（300 mg BID）28天，联合口服环丙沙星（15-20 mg/kg BID）或口服安慰剂14天。试验的主要终点是至需要IV抗生素治疗的肺部加重或因呼吸道症状住院的时间，以及研究期间新发Pa阳性呼吸道培养的患者比例。观察性研究的广泛目标是描述与早期获得Pa相关的风险因素和结局。306例患者被随机分配在临床试验和1787人参加了队列study.Conclusions：这些同伴研究将提供有价值的流行病学和微生物学信息的早期CIF肺疾病和PA收购，安全性和临床疗效数据的抗假单胞菌治疗策略的早期PA感染的婴幼儿CF的气道。(C)2009 Elsevier Inc. All rights reserved.
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.