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Treatment evolution in high-risk congenital diaphragmatic hernia: ten years' experience with diaphragmatic agenesis
Kevin P Lally1, Pamela A Lally, Krisa P Van Meurs
1Department of Surgery, University of Texas Medical School Houston and Memorial Hermann Children's Hospital, Houston, TX, USA. kevin.p.lally@uth.tmc.edu
Insights
Newer therapies for congenital diaphragmatic hernia (CDH) show promise, with less reliance on ECMO and more use of inhaled nitric oxide (iNO) in high-risk infants. Survival trends improved, but challenges in agenesis patients persist.
Area of Science:
- Neonatal surgery
- Pediatric critical care
- Congenital abnormalities
Background:
- Congenital diaphragmatic hernia (CDH) is a major cause of neonatal mortality.
- Infants with large defects or diaphragmatic agenesis face the highest mortality and morbidity risks.
- Novel therapeutic interventions are increasingly utilized in managing CDH.
Purpose of the Study:
- To assess the impact of emerging therapies on high-risk congenital diaphragmatic hernia (CDH) patients.
- Specifically evaluate outcomes in neonates with diaphragmatic agenesis.
- Analyze treatment trends and survival rates over a decade.
Main Methods:
- Prospective data collection from 20 centers across 5 countries over 10 years.
- Inclusion of all liveborn infants diagnosed with CDH.
- Follow-up until patient death or hospital discharge.
Main Results:
- Overall survival for 1,569 CDH patients was 68%; survival for 218 patients with diaphragmatic agenesis was 54%.
- Between the study's early and late periods, use of ECMO decreased (75% to 52%) while iNO use increased (30% to 80%) in agenesis patients.
- A trend towards improved survival for agenesis patients was observed (47% to 59%), alongside prolonged hospital stays and significant morbidity (tube feeding, oxygen therapy).
Conclusions:
- Management of CDH has shifted towards reduced ECMO use and increased iNO use in high-risk cases, potentially improving survival.
- Despite therapeutic advancements, mortality, prolonged hospital stays, and morbidity remain significant concerns for infants with diaphragmatic agenesis.
- Further research is needed to optimize outcomes for the most critically ill CDH patients.
Objective:
The objective of this study was to evaluate the impact of newer therapies on the highest risk patients with congenital diaphragmatic hernia (CDH), those with agenesis of the diaphragm.
Summary Background Data:
CDH remains a significant cause of neonatal mortality. Many novel therapeutic interventions have been used in these infants. Those children with large defects or agenesis of the diaphragm have the highest mortality and morbidity.
Methods:
Twenty centers from 5 countries collected data prospectively on all liveborn infants with CDH over a 10-year period. The treatment and outcomes in these patients were examined. Patients were followed until death or hospital discharge.
Results:
A total of 1,569 patients with CDH were seen between January 1995 and December 2004 in 20 centers. A total of 218 patients (14%) had diaphragmatic agenesis and underwent repair. The overall survival for all patients was 68%, while survival was 54% in patients with agenesis. When patients with diaphragmatic agenesis from the first 2 years were compared with similar patients from the last 2 years, there was significantly less use of ECMO (75% vs. 52%) and an increased use of inhaled nitric oxide (iNO) (30% vs. 80%). There was a trend toward improved survival in patients with agenesis from 47% in the first 2 years to 59% in the last 2 years. The survivors with diaphragmatic agenesis had prolonged hospital stays compared with patients without agenesis (median, 68 vs. 30 days). For the last 2 years of the study, 36% of the patients with agenesis were discharged on tube feedings and 22% on oxygen therapy.
Conclusions:
There has been a change in the management of infants with CDH with less frequent use of ECMO and a greater use of iNO in high-risk patients with a potential improvement in survival. However, the mortality, hospital length of stay, and morbidity in agenesis patients remain significant.
