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严重支气管肺发育不良患儿的肺功能测试和表型。

Infant Pulmonary Function Testing and Phenotypes in Severe Bronchopulmonary Dysplasia.

机构信息

Comprehensive Center for Bronchopulmonary Dysplasia and the Neonatal Aerodigestive Program, and.

Department of Pediatrics, The Ohio State University, Columbus, Ohio.

出版信息

Pediatrics. 2018 May;141(5). doi: 10.1542/peds.2017-3350. Epub 2018 Apr 5.

Abstract

BACKGROUND

The definition of severe bronchopulmonary dysplasia (sBPD) is based on respiratory support needs. The management of a patient with sBPD remains empirical and is highly variable among providers. Our objective in this study was to test the hypothesis that infant pulmonary function testing (iPFT) would reveal distinct phenotypes in patients with established sBPD during the initial NICU stay.

METHODS

A prospective cohort study with data prospectively collected on infants with sBPD from May 1, 2003, to June 30, 2016. iPFT data were used to classify the patients as obstructive, restrictive, or mixed.

RESULTS

The median gestational age at birth was 25 weeks (interquartile range [IQR], 24-27 weeks) and the median birth weight was 707 g (IQR, 581-925 g). At the time of iPFT, the median postmenstrual age was 52 weeks (IQR, 45-63 weeks), and the median weight was 4.4 kg (IQR, 3.7-6.0 kg). There were 56 (51%) patients with obstructive, 44 (40%) with mixed, and 10 (9%) with restrictive phenotypes. Moderate or severe obstruction was seen in 86% of the obstructive group and 78% of the mixed group. Of the restrictive patients, 70% had moderate and 30% had mild restriction. Bronchodilator response was seen in 74% of obstructive, 63% of mixed, and 25% of restrictive patients.

CONCLUSIONS

Our findings reveal that sBPD as it is currently defined includes distinct phenotypes. Future researchers of diagnostic approaches to this population should consider the development of bedside tests to define phenotypes, and researchers in future therapeutic trials should consider the use of pulmonary function phenotyping in patient recruitment.

摘要

背景

严重支气管肺发育不良(sBPD)的定义基于呼吸支持需求。sBPD 患者的管理仍然是经验性的,并且在提供者之间差异很大。我们在这项研究中的目的是检验以下假设,即在新生儿重症监护病房(NICU)住院期间,对已确诊的 sBPD 患者进行婴儿肺功能测试(iPFT)将揭示出明显的表型。

方法

这是一项前瞻性队列研究,对 2003 年 5 月 1 日至 2016 年 6 月 30 日期间患有 sBPD 的婴儿进行前瞻性数据收集。使用 iPFT 数据将患者分类为阻塞性、限制性或混合性。

结果

中位胎龄为 25 周(四分位距 [IQR],24-27 周),中位出生体重为 707g(IQR,581-925g)。进行 iPFT 时,中位校正胎龄为 52 周(IQR,45-63 周),中位体重为 4.4kg(IQR,3.7-6.0kg)。56 例(51%)患者表现为阻塞性、44 例(40%)为混合性、10 例(9%)为限制性表型。86%的阻塞组和 78%的混合组存在中度或重度阻塞。70%的限制性患者存在中度限制,30%存在轻度限制。在阻塞组中,74%、混合组中 63%和限制性组中 25%的患者对支气管扩张剂有反应。

结论

我们的发现表明,目前定义的 sBPD 包括明显的表型。未来针对该人群的诊断方法的研究人员应考虑开发床边测试来定义表型,而未来治疗试验的研究人员应考虑在患者招募中使用肺功能表型。

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