Family-focused, family-driven research to improve care and outcomes: 3 Questions with Samir Gadepalli

What is the impact of this condition, and what do we know about prevention and treatment?

NEC is the most common and catastrophic intestinal disease among premature infants, who account for about 80 percent of diagnoses. NEC affects 1-3 per 1000 live births, or about 9,000 infants each year in the U.S. With this disease, intestinal tissue becomes inflamed and dies off, which can lead to infections and many other life-threatening complications. It’s a major cause of mortality and morbidity in neonatal intensive care units (NICUs), where NEC cases make up 1-7 percent of all admissions.

Little is known about how to prevent this condition, or how to predict which infants will develop it beyond known risk factors such as low birth weight and prematurity. Once NEC has been identified, treatment is often complex and intensive, but there’s also huge variation in treatment protocols. Infants who survive NEC often have lifelong neurological and nutritional complications, which can have a devastating impact on families and their long-term well-being.

How does improving outcomes for this condition depend on families?

Families drive progress. Some of the outcomes that we care about as clinicians or researchers, such as time spent in a hospital or days on a ventilator, are meaningless to families who are left to manage the disease and its consequences long after they leave the hospital setting. As with many other serious childhood health conditions, quality of life measures are only truly useful and informative if they consider the broad effects of an illness on family well-being. So that means looking at family-level outcomes, not just patient-level outcomes. The families responsible for caring for these children are the only ones who can inform us about some of the critical factors to consider in research that looks at how to improve life for these kids. We also lack the large, long-term follow-up studies needed to measure long-term economic outcomes of NEC like the costs of healthcare and social and educational services, and families need to be central in shaping that work and determining research priorities in general.

How is the NEC Society working to drive research and family-centered care forward?

This group brings together clinicians and researchers from around the world, but it was founded by and is led by families affected by NEC. The Society launched the first Research Collaborative for NEC, with more than 40 investigators from dozens of institutions participating in joint research ventures. This network is kind of a mix of basic science and health services research that is guided by the knowledge and experiences of NEC patients and their families.

One project underway is the establishment of a national benchmarking database for NICUs in institutions that are part of the Collaborative. This will allow us to analyze the variation of care and compliance to protocols for NEC, information that’s intended to help develop recommended standards of care. We want to look not just at medical outcomes, but social and family outcomes – and so NEC families have been involved from the beginning in the planning and implementation of this assessment to make sure we’re considering all the factors that may have an impact.

The NEC Collaborative has also established a unified biorepository that will help with prospective collection of biologic samples linked with clinical data that can be used by the Collaborative to achieve faster scientific progress.

The Patient-Centered Outcomes Research Institute (PCORI) has also supported two NEC Society symposia to bring families, clinicians and researchers together to chart the course for advancing prevention and treatment of this disease, with the 2019 Symposium coming up June 2-5 in Ann Arbor (NICHD is also a sponsor of this symposium).