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支气管肺发育不良发现(D-BPD)项目:一项前瞻性转化多中心合作研究的研究方案,旨在调查极低出生体重儿慢性肺病的决定因素。

The discovery BPD (D-BPD) program: study protocol of a prospective translational multicenter collaborative study to investigate determinants of chronic lung disease in very low birth weight infants.

作者信息

Ofman Gaston, Caballero Mauricio T, Alvarez Paggi Damian, Marzec Jacqui, Nowogrodzki Florencia, Cho Hye-Youn, Sorgetti Mariana, Colantonio Guillermo, Bianchi Alejandra, Prudent Luis M, Vain Nestor, Mariani Gonzalo, Digregorio Jorge, Turconi Elba Lopez, Osio Cristina, Galletti Fernanda, Quiros Mariangeles, Brum Andrea, Lopez Garcia Santiago, Garcia Silvia, Bell Douglas, Jones Marcus H, Tipple Trent E, Kleeberger Steven R, Polack Fernando P

机构信息

Department of Pediatrics, University of Alabama at Birmingham, Birmingham, USA.

Fundación INFANT, Buenos Aires, Argentina.

出版信息

BMC Pediatr. 2019 Jul 6;19(1):227. doi: 10.1186/s12887-019-1610-8.

Abstract

BACKGROUND

Premature birth is a growing and serious public health problem affecting more than one of every ten infants worldwide. Bronchopulmonary dysplasia (BPD) is the most common neonatal morbidity associated with prematurity and infants with BPD suffer from increased incidence of respiratory infections, asthma, other forms of chronic lung illness, and death (Day and Ryan, Pediatr Res 81: 210-213, 2017; Isayama et la., JAMA Pediatr 171:271-279, 2017). BPD is now understood as a longitudinal disease process influenced by the intrauterine environment during gestation and modulated by gene-environment interactions throughout the neonatal and early childhood periods. Despite of this concept, there remains a paucity of multidisciplinary team-based approaches dedicated to the comprehensive study of this complex disease.

METHODS

The Discovery BPD (D-BPD) Program involves a cohort of infants < 1,250 g at birth prospectively followed until 6 years of age. The program integrates analysis of detailed clinical data by machine learning, genetic susceptibility and molecular translation studies.

DISCUSSION

The current gap in understanding BPD as a complex multi-trait spectrum of different disease endotypes will be addressed by a bedside-to-bench and bench-to-bedside approach in the D-BPD program. The D-BPD will provide enhanced understanding of mechanisms, evolution and consequences of lung diseases in preterm infants. The D-BPD program represents a unique opportunity to combine the expertise of biologists, neonatologists, pulmonologists, geneticists and biostatisticians to examine the disease process from multiple perspectives with a singular goal of improving outcomes of premature infants.

TRIAL REGISTRATION

Does not apply for this study.

摘要

背景

早产是一个日益严重的公共卫生问题,影响着全球每十名婴儿中的不止一名。支气管肺发育不良(BPD)是与早产相关的最常见的新生儿疾病,患有BPD的婴儿呼吸道感染、哮喘、其他形式的慢性肺病和死亡的发生率增加(戴和瑞安,《儿科学研究》81:210 - 213,2017;稻山等人,《美国医学会儿科学杂志》171:271 - 279,2017)。现在认为BPD是一种纵向疾病过程,受孕期子宫内环境影响,并在新生儿期和幼儿期受基因 - 环境相互作用调节。尽管有这一概念,但仍然缺乏致力于对这种复杂疾病进行综合研究的多学科团队方法。

方法

发现BPD(D - BPD)项目纳入了一组出生时体重<1250克的婴儿,前瞻性随访至6岁。该项目整合了通过机器学习、遗传易感性和分子转化研究对详细临床数据的分析。

讨论

D - BPD项目将通过床边到实验台再到床边的方法解决目前将BPD理解为不同疾病终型的复杂多特征谱方面的差距。D - BPD将增强对早产儿肺部疾病的机制、演变和后果的理解。D - BPD项目提供了一个独特的机会,将生物学家、新生儿科医生、肺科医生、遗传学家和生物统计学家的专业知识结合起来,从多个角度检查疾病过程,唯一目标是改善早产儿的结局。

试验注册

本研究不适用。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e7a8/6612113/6807852cba11/12887_2019_1610_Fig1_HTML.jpg

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