Breaking Down the Barriers of NEC Prevention and Treatment

项目总结 NEC研讨会的目标是推动NEC社区改善预防和治疗的能力 通过以患者为中心的结局研究和临床比较，为有NEC风险的脆弱婴儿提供选择 有效性研究。这次会议的愿景是激发新的项目、研究和实践变化 通过多学科合作，开始从根本上改善脆弱的婴儿和他们的 家庭在NEC诊断之前、期间和之后接受护理。NEC应该得到适当的优先顺序 资源和关注，因为这是一种可能导致败血症的灾难性肠道疾病，1 器官衰竭(包括肾脏、脑和肺)，手术切除所致的短肠综合征，需要长时间- 长期静脉营养会导致肝脏损伤，3人甚至死亡。NEC在美国的死亡率 低出生体重人口(1500克)的比例在15%-42%之间。4而早产儿最多 易感，无法预测哪些婴儿会患上NEC，以及他们的破坏性 疾病过程。因此，家庭和临床医生对NEC的诊断是盲目和沮丧的，因为 早期发现的工具和预防战略不存在或不发达， 机构在喂养方法、输血指南、供体母乳的可获得性或使用 益生菌。在NEC首次发病后存活下来的婴儿通常会遭受终身神经和营养问题的困扰 并发症。虽然NEC给家庭带来的社会和情感代价是不可估量的，但 据估计，美国每年的NEC约为30亿美元。NEC研讨会将开始 通过召集一些最坚定的NEC患者来应对这种复杂疾病带来的这些挑战 来自世界各地的倡导者、提供者和科学家。研讨会是通过一个 NEC协会和密歇根大学儿科外科之间的合作伙伴关系， 来自以患者为中心的结果研究所颁发的参与奖的支持。美国国家经济委员会 研讨会的目标包括：1)推动由NEC协会推动的现有和新的研究项目 通过整合研讨会会议和协作研究目标实现协作研究；2) 为NICU开发“NEC预防工具包”，以支持使用有关NEC的通用最佳做法 预防、改善与NEC相关的患者/提供者沟通、NEC意识和患者家属 决策；以及3)建立国家经济共同体研究协作组的能力，以实现变革和组织振兴/经济、社会和文化权利的目标 优先事项。在NEC研讨会举行后，NEC学会研究合作，一个分支机构 由50名多学科科学家组成的NEC协会将实施这些项目和传播计划 来自会议的消息。NEC研讨会的预期成果包括：1)推动 生物标志物、治疗和干预；2)探索治疗和治疗的有效性和有效性 干预措施；以及3)在NEC协会代表的中心实施和传播研究。

PROJECT SUMMARY The goal of the NEC Symposium is to propel the NEC community's ability to improve prevention and treatment options for fragile infants at risk of NEC through patient-centered outcomes research and comparative clinical effectiveness research. The vision for this meeting is to spark new projects, research, and practice changes through multi-disciplinary collaborations that begin to fundamentally improve how vulnerable infants and their families receive care before, during, and after the NEC diagnosis. NEC deserves adequate prioritization of resources and attention, given that it is a catastrophic intestinal disease that can result in sepsis,1 multiple organ failure (including kidneys,2 brain and lungs), short gut syndrome from surgical resections requiring long- term intravenous nutrition with subsequent liver damage,3 and even death. Mortality rates for NEC in the very low birth weight population (<1500 grams) range from 15-42%.4 While premature neonates are the most susceptible, it is impossible to predict which infants will develop NEC and the devastating nature of their disease process. Accordingly, families and clinicians are blindsided and frustrated by the diagnosis of NEC, as tools for early detection and strategies for prevention are non-existent or underdeveloped, with tremendous variation at institutions in feeding approaches, transfusion guidelines, availability of donor milk, or the use of probiotics. Infants that survive the initial onset of NEC often suffer with lifelong neurological and nutritional complications. While the social and emotional costs of NEC to families is immeasurable, the economic costs of NEC are estimated to be approximately $3 billion per year in the U.S.5 The NEC Symposium will begin to address these challenges from this complex disease by convening some of the most committed NEC patient advocates, providers and scientists from around the world. The Symposium is made possible through a partnership between the NEC Society and the University of Michigan’s Section of Pediatric Surgery, with support from an Engagement Award from the Patient-Centered Outcomes Research Institute. The NEC Symposium’s objectives include: 1) propel current and new research projects driven by the NEC Society Research Collaborative through the integration of Symposium sessions and Collaborative research aims; 2) develop a “NEC Prevention Toolkit” for NICUs to support the use of universal best practices regarding NEC prevention, improve NEC-related Patient/Provider Communication, NEC awareness, and Patient-Family decision-making; and 3) build the capacity of the NEC Society Research Collaborative to pursue PCOR/CER priorities. After the NEC Symposium takes place, the NEC Society Research Collaborative, a branch of the NEC Society comprised of 50 multidisciplinary scientists, will carry out the projects and dissemination plans stemming from the meeting. Anticipated outcomes of the NEC Symposium include: 1) drive the development of biomarkers, treatments and interventions; 2) explore the efficacy and effectiveness of treatments and interventions; and 3) implement and disseminate research in centers represented by the NEC Society.