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Published on: March 12, 2020
Congenital diaphragmatic hernia. Epidemiology and outcome
M R Langham1, D W Kays, D J Ledbetter
1Department of Surgery, University of Florida, College of Medicine, Gainesville, USA. langham@surgery.ufl.edu
Insights
Congenital diaphragmatic hernia (CDH) affects many newborns, with survival rates around 60%. Further research is needed to understand its causes and improve treatment strategies for this serious birth defect.
Area of Science:
- Pediatric Surgery
- Neonatology
- Birth Defects Research
Background:
- Congenital diaphragmatic hernia (CDH) is a significant birth defect impacting approximately 1114 infants annually in the U.S.
- Survival rates for CDH average 60%, with considerable institutional and geographical variability.
- The etiology of CDH remains largely unknown, with evidence suggesting multiple causative factors due to its association with other anomalies and diverse presentations.
Purpose of the Study:
- To address the lack of comparative data on CDH treatment efficacy.
- To establish a framework for stratifying CDH patients into more homogeneous groups for clinical trials.
- To initiate a multicenter data collection effort to facilitate research on CDH.
Main Methods:
- Formation of the Congenital Diaphragmatic Hernia Study Group, a multicenter collaborative.
- Initiation of prospective data collection to gather comprehensive information on CDH cases.
- Development of a patient stratification scheme as an initial research goal.
Main Results:
- CDH is a leading cause of perinatal morbidity and mortality.
- Significant variability exists in CDH treatment approaches and patient outcomes.
- Current data limitations hinder rigorous comparison of different treatment strategies.
Conclusions:
- CDH necessitates further investigation into its etiology and management.
- Multicenter collaboration is essential for conducting meaningful clinical trials in CDH.
- Stratification of CDH patients is a critical step toward improving treatment efficacy and outcomes.
Abstract:
Congenital diaphragmatic hernia is a relatively common birth defect. It affects about 1114 babies a year in the United States. Reported survival averages 60% but may be significantly lower. We do not understand the etiology of CDH. Its association with other anomalies and several distinct patterns of presentation suggest that more than one cause may exist. There is a high degree of variability in both treatment and outcomes, but no data exist to allow a rigorous comparison of the efficacy of various treatment strategies. Stratification of patients into more homogeneous groups will be a necessary prerequisite for the design of meaningful comparative trials. The incidence of the lesion prevents any single institution from accruing sufficient patients to conduct such a trial. An ad hoc multicenter study group (the Congenital Diaphragmatic Hernia Study Group) has been formed for this purpose. This organization has begun collecting data with an initial goal of developing a stratification scheme. Prospective data collection should allow verification of several of the estimates made in this article. Current data make it clear that CDH represents a major cause of perinatal morbidity and mortality.
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