When it comes to necrotizing enterocolitis (NEC), there are so many unanswered questions and very few resources. Our multidisciplinary team undertook a two-year project to identify the most urgent and impactful research topics to help our community decide where to direct the limited funding and resources available for NEC research.

Over the course of this project, 11 patient-families personally affected by NEC and 11 clinician-scientists formed a team. They worked together to identify the most pressing and important patient-centered research questions in the NEC field.

The team first identified over 150 potential research topics, and collaboratively narrowed these to 20 prioritized topics, using the Delphi method. The full process will be formally published in the coming months.

This project was made possible by an Engagement Award from the Patient-Centered Outcomes Research Institute (PCORI), and support from the Chan Zuckerberg Initiatve.

Researchers, we hope you will join us in taking up these patient-centered research topics!

Additionally, we have compiled our lessons learned from this project into a guide for other rare disease communities. We hope our experience will be useful to you as you undertake you own research prioritization project. Click on the image below to read our guide, “Building a Prioritized Research Agenda: Lessons from the NEC Society.”

Click here to read the guide

Full List of NEC Patient-Centered Research Priorities

(Summarized in photo graphic above)
TOPIC 1: Development and use of medications (e.g. novel or existing such as lactoferrin or probiotics) or treatments to prevent NEC
TOPIC 2: Optimizing the use of human milk (mother’s own milk, donor milk, fresh vs. frozen, colostrum, components such as oligosaccharides) and lactation support for prevention of NEC
TOPIC 3: Tools, such as clinical scoring, biomarkers (e.g. blood tests), or imaging (e.g. ultrasound), for diagnosis of NEC
TOPIC 4: Acute medical treatment and management of NEC, including novel treatments, biomarkers, imaging, antibiotic approaches, re-introduction of feeding, nursing care, treatment of pain and avoiding brain injury.
TOPIC 5: Patient-family engagement, empowerment, and integration into the NICU care-team, including role of parents and partnership with clinicians in the timeliness of NEC diagnosis
TOPIC 6: Long-term gastrointestinal outcomes, including short bowel and other non-neurodevelopmental health outcomes, from NEC that include related quality-of-life measures
TOPIC 7: Quality Improvement for NEC
TOPIC 8: Identifying practices that increase NEC risk
TOPIC 9: Long-term non-GI outcomes and fertility, and related quality of life measures among NEC survivors
TOPIC 10: Communication amongst the healthcare team and patient-families before and after NEC and improving actionable responses to parent concerns
TOPIC 11: Proactive approaches to support patient-families with follow-up care and decisions,
including developmental and intestinal failure care
TOPIC 12: Chronic treatment and recovery of NEC, including subsequent surgeries, intestinal rehabilitation and transplant, as well as development of strategies to protect the liver and brain, and supporting transitions of care (e.g. to home, to adult medicine).
TOPIC 13: Educational approaches on NEC for patients and providers, before and after NEC
TOPIC 14: Impact of nutritional composition and approaches (enteral and parenteral)
including type, initiation, speed of advancement, standardized regimen and feeding during transfusion on occurrence of NEC
TOPIC 15: Optimizing informed choice and shared-decision making, including ethical issues, in NEC
TOPIC 16: Fortification of human milk
TOPIC 17: Initial surgical treatment of NEC
TOPIC 18: Acute and long-term mental health of patient-families impacted by NEC
TOPIC 19: Financial and social-emotional burdens of NEC for patient-families, healthcare providers, hospitals and society
TOPIC 20: Disparities in NEC incidence and outcomes and inequity in decision-making and access to care and services (e.g. palliative care team)