Queensland Children's Medical Research Institute, and Queensland Children's Respiratory Centre, Royal Children's Hospital, University of Queensland, Brisbane, Australia.
JAMA. 2011 Jul 13;306(2):163-71. doi: 10.1001/jama.2011.954.
Early pulmonary infection in children with cystic fibrosis leads to increased morbidity and mortality. Despite wide use of oropharyngeal cultures to identify pulmonary infection, concerns remain over their diagnostic accuracy. While bronchoalveolar lavage (BAL) is an alternative diagnostic tool, evidence for its clinical benefit is lacking.
To determine if BAL-directed therapy for pulmonary exacerbations during the first 5 years of life provides better outcomes than current standard practice relying on clinical features and oropharyngeal cultures.
DESIGN, SETTING, AND PARTICIPANTS: The Australasian Cystic Fibrosis Bronchoalveolar Lavage (ACFBAL) randomized controlled trial, recruiting infants diagnosed with cystic fibrosis through newborn screening programs in 8 Australasian cystic fibrosis centers. Recruitment occurred between June 1, 1999, and April 30, 2005, with the study ending on December 31, 2009.
BAL-directed (n = 84) or standard (n = 86) therapy until age 5 years. The BAL-directed therapy group underwent BAL before age 6 months when well, when hospitalized for pulmonary exacerbations, if Pseudomonas aeruginosa was detected in oropharyngeal specimens, and after P. aeruginosa eradication therapy. Treatment was prescribed according to BAL or oropharyngeal culture results.
Primary outcomes at age 5 years were prevalence of P. aeruginosa on BAL cultures and total cystic fibrosis computed tomography (CF-CT) score (as a percentage of the maximum score) on high-resolution chest CT scan.
Of 267 infants diagnosed with cystic fibrosis following newborn screening, 170 were enrolled and randomized, and 157 completed the study. At age 5 years, 8 of 79 children (10%) in the BAL-directed therapy group and 9 of 76 (12%) in the standard therapy group had P. aeruginosa in final BAL cultures (risk difference, -1.7% [95% confidence interval, -11.6% to 8.1%]; P = .73). Mean total CF-CT scores for the BAL-directed therapy and standard therapy groups were 3.0% and 2.8%, respectively (mean difference, 0.19% [95% confidence interval, -0.94% to 1.33%]; P = .74).
Among infants diagnosed with cystic fibrosis, BAL-directed therapy did not result in a lower prevalence of P. aeruginosa infection or lower total CF-CT score when compared with standard therapy at age 5 years.
anzctr.org.au Identifier: ACTRN12605000665639.
儿童囊性纤维化的早期肺部感染会导致发病率和死亡率增加。尽管广泛使用咽拭子培养来识别肺部感染，但人们仍然对其诊断准确性存在担忧。虽然支气管肺泡灌洗（BAL）是一种替代的诊断工具，但缺乏其临床获益的证据。
确定在生命的前 5 年，针对肺部恶化进行 BAL 指导的治疗是否比目前依赖临床特征和咽拭子培养的标准治疗提供更好的结果。
设计、地点和参与者：澳大利亚囊性纤维化支气管肺泡灌洗（ACFBAL）随机对照试验，在 8 个澳大利亚囊性纤维化中心的通过新生儿筛查计划诊断为囊性纤维化的婴儿中招募。招募工作于 1999 年 6 月 1 日至 2005 年 4 月 30 日进行，研究于 2009 年 12 月 31 日结束。
BAL 指导（n = 84）或标准（n = 86）治疗至 5 岁。BAL 指导治疗组在 6 个月大之前、因肺部恶化住院时、咽拭子标本中检测到铜绿假单胞菌时、以及在铜绿假单胞菌根除治疗后，进行 BAL。根据 BAL 或咽拭子培养结果进行治疗。
5 岁时的主要结局是 BAL 培养中铜绿假单胞菌的流行率和高分辨率胸部 CT 扫描上的总囊性纤维化 CT 评分（以最大评分的百分比表示）。
在通过新生儿筛查诊断出的 267 名囊性纤维化婴儿中，有 170 名被纳入并随机分组，其中 157 名完成了研究。在 5 岁时，BAL 指导治疗组中有 8 名（10%）儿童和标准治疗组中有 9 名（12%）儿童的最终 BAL 培养中存在铜绿假单胞菌（风险差异，-1.7%[95%置信区间，-11.6%至 8.1%]；P =.73）。BAL 指导治疗组和标准治疗组的平均总 CF-CT 评分分别为 3.0%和 2.8%（平均差异，0.19%[95%置信区间，-0.94%至 1.33%]；P =.74）。
在囊性纤维化婴儿中，与标准治疗相比，BAL 指导治疗在 5 岁时并未导致铜绿假单胞菌感染的发生率降低或总 CF-CT 评分降低。
anzctr.org.au 标识符：ACTRN12605000665639。
