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Cost-effectiveness analysis of extended ECMO duration in newborns with congenital diaphragmatic hernia in the United States

Congenital diaphragmatic hernia (CDH) is a serious and sometimes fatal congenital diaphragmatic abnormality.

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Congenital diaphragmatic hernia (CDH) is a serious and sometimes fatal congenital diaphragmatic abnormality. The abdominal contents migrate into the pleural cavity, and the deficiency is accompanied by a range of lung and pulmonary vascular bed maldevelopment. Advanced life-support methods, such as extracorporeal membrane oxygenation (ECMO), are frequently required in patients with increasingly severe diseases. According to the Extracorporeal Life Support Registry, newborns with CDH who require ECMO have a 50% chance of survival. ECMO is a type of temporary heart-lung bypass that allows patients to be stabilized while awaiting surgical treatment. Patients who fit the ECMO criteria can be kept alive for a long time if their pulmonary and cardiac functions improve. The current research aimed to use current data to predict the cost-effectiveness of longer ECMO duration in CDH newborns who might benefit from more than two weeks of ECMO.

In CDH neonates who may benefit from prolonged ECMO duration, a decision tree model was constructed from a societal perspective in the United States to assess the cost-effectiveness of (1) ECMO duration of 2 weeks, (2) ECMO length of 3 weeks, and (3) ECMO duration >3 weeks. The patient's costs and quality-adjusted life-years (QALYs) were discounted by 3% annually to the patient's birth year. The most important long-term outcomes with quantifiable clinical and economic implications over the lifespan of the affected survivors, such as lung dysfunction and irreversible neurocognitive deficits, were considered, and they were stratified by severity. Because cognitive disability has a significant impact on life expectancy, survivors were expected to live a full life at release, depending on the degree of their neurological issues.

Sensitivity analyses yielded outcomes that were similar to those of the base case analysis. For all three comparisons in the model, one-way sensitivity tests revealed that the chance of survival at discharge, followed by first encounter costs and the probability of severe neurological damage, had the biggest influence on the observed incremental NMB. Despite advancements in other ventilatory techniques and wider use of nitric oxide and other pulmonary vasodilators, ECMO continues to play an important role in the care of CDH newborns. ECMO has become a prominent target for CEA study because it is a life-saving therapy that is also costly and intrusive. For babies with respiratory failure, especially those with CDH, several studies have been undertaken to compare the cost-effectiveness of ECMO to conventional or other creative alternative therapies.

PHIS dataset was utilized to estimate the probability of long-term sequelae using proxy diagnoses at discharge for pulmonary and compromised neurological outcomes, which may not be a real picture of the long-term repercussions suffered by the survivors due to a lack of evidence on long-term follow-up. In the model, only pulmonary dysfunction and neurocognitive impairment were thought to have a long-term influence on survivors. Given escalating healthcare costs and limited resources, economic ramifications are just one of many factors that might influence social decisions. To further guide professional decision-making and family counseling in the neonatal intensive care unit, more research on the association between ECMO duration and long-term outcomes and sequelae of CDH patients post-discharge is needed.

Read more:
Zheng H, Gong C, Chapman R, Yieh L, Friedlich P, Hay JW. Cost-effectiveness analysis of extended extracorporeal membrane oxygenation duration in newborns with congenital diaphragmatic hernia in the United States. Pediatr Neonatol. 2021 Oct 18:S1875-9572(21)00202-3. doi: 10.1016/j.pedneo.2021.08.015. Epub ahead of print. PMID: 34742677.

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