1 Department of Pediatrics, Children's Hospital of Richmond at Virginia Commonwealth University, Richmond, Virginia.
2 Department of Pediatrics, Case Western Reserve University School of Medicine, Cleveland, Ohio.
Ann Am Thorac Soc. 2018 Feb;15(2):225-233. doi: 10.1513/AnnalsATS.201702-111OC.
There are important gaps in knowledge of the optimal treatment of cystic fibrosis pulmonary exacerbations. Previous observational studies comparing inpatient with outpatient treatment have suffered from methodologic weaknesses, especially indication bias.
We analyzed data from the Epidemiologic Study of Cystic Fibrosis using techniques to control for indication bias to determine whether there is an advantage to inpatient treatment of cystic fibrosis pulmonary exacerbations.
We identified typical pulmonary exacerbations in patients ages 6 years and older during the 3-year observation period ending in 2005. In our primary analysis, we used the instrumental variables method, implemented using two-stage least squares regression, to evaluate the effect of the proportion of total time that intravenous treatment was administered on an inpatient (versus outpatient) basis on the likelihood of return of percent predicted forced expiratory volume in 1 second to greater than or equal to 90% of baseline post-treatment. We also evaluated two other indicators of treatment setting, three other measures of treatment response, and two alternative modeling techniques, and we also looked for differences between children and adults.
Our final analysis included 4,497 pulmonary exacerbations in 2,773 individual patients at 75 sites. We calculated the mean proportion of intravenous treatment time that was provided in the hospital setting at each site. The median across sites was 0.581 (interquartile range, 0.396-0.753). The median treatment success rate across sites was 74.2% (interquartile range, 67.9 to 79.2%). Univariate analysis and two-stage least squares models showed a positive relationship between treatment success and proportion of inpatient treatment days. Our primary model revealed an absolute increase of 9.08% (95% confidence interval, 2.55-15.61; P = 0.006) in the achievement of a return of percent predicted forced expiratory volume in 1 second to greater than or equal to 90% of baseline comparing complete inpatient treatment with no inpatient treatment. Treatment response was not related to duration of intravenous therapy. Similar results were found for all our modeling techniques and outcomes.
Patients with cystic fibrosis treated at sites with more reliance on inpatient treatment were more likely to achieve successful forced expiratory volume in 1 second recovery. There was no relationship between treatment duration and recovery of forced expiratory volume in 1 second.
在囊性纤维化肺部恶化的最佳治疗方法方面，我们的知识还存在重要的空白。先前比较住院和门诊治疗的观察性研究受到方法学的局限，尤其是适应证偏倚。
我们使用控制适应证偏倚的技术分析囊性纤维化的流行病学研究的数据，以确定住院治疗囊性纤维化肺部恶化是否具有优势。
我们在 2005 年结束的 3 年观察期内识别出年龄在 6 岁及以上患者的典型肺部恶化。在我们的主要分析中，我们使用了工具变量法，通过两阶段最小二乘法回归实施，以评估静脉治疗的总时间中以住院（而非门诊）为基础的比例对治疗后 1 秒用力呼气量恢复到大于或等于基线的 90%的可能性的影响。我们还评估了治疗方案的另外两个指标、治疗效果的另外三个衡量指标和两种替代建模技术，并且我们还观察了儿童和成人之间的差异。
我们的最终分析包括来自 75 个地点的 2773 名患者的 4497 次肺部恶化。我们计算了每个地点提供的住院治疗时间的平均比例。各个地点的中位数为 0.581（四分位距，0.396-0.753）。各个地点的中位治疗成功率为 74.2%（四分位距，67.9 至 79.2%）。单变量分析和两阶段最小二乘法模型显示，治疗成功率与住院治疗天数之间存在正相关关系。我们的主要模型显示，与完全门诊治疗相比，门诊治疗的绝对增加了 9.08%（95%置信区间，2.55-15.61；P=0.006），从而使 1 秒用力呼气量恢复到大于或等于基线的 90%。治疗效果与静脉治疗时间无关。我们所有的建模技术和结果都得到了类似的结果。
在更多地依赖住院治疗的地点接受治疗的囊性纤维化患者更有可能成功地恢复 1 秒用力呼气量。治疗持续时间与 1 秒用力呼气量的恢复无关。
