Neonatal Anemia and Thrombocytopenia: Pathophysiology and Treatment

此次PPG更新的统一主题是了解危重早产儿最严重和最常见的两种疾病的病理生理学，并建立最佳治疗方案：贫血和血小板减少。目前对这些疾病的治疗在很大程度上依赖于红细胞和血小板的输注，这些治疗方法仍然存在争议，研究也不完全。我们的PPG小组和其他人之前的研究已经证实，早产儿贫血和血小板减少的许多病理生理学方面是独一无二的。然而，我们的理解是不完整的。因此，我们更新的目标是进一步确定导致这些紧迫和相关临床条件的机制，根据这些机制知识优化它们的治疗，从而减少与这些条件相关的短期和长期残疾。我们的四个相互关联的项目，其目标与我们的统一主题直接相关，包括：项目1“优化促红细胞生成素治疗新生儿贫血”，PL JA Widness(和P Veng-Pedersen)；项目2“新生儿血小板减少的发病机制和治疗”，PL M Sola-Visner；项目3“早产输血：脑结构/功能结果”，PL PC Nopoulos；以及项目4“新生儿贫血在学习和记忆中的作用”，PL MK Georgiff。核心A(“行政、统计和研究人员”)和核心B(“实验室和数据库”，PL DM Mock)将为所有项目提供支持。为了提高有效性，我们的计划扩大到包括四个(而不是两个)表现站点，因此数据可以在项目1、2和3之间共享(即来自相同婴儿的数据将在多个站点的研究中使用)。我们成功进行多站点研究的历史将在协调不同性能站点的研究方面发挥无价的作用。由于我们的计划依赖于一个由新的和有经验的研究人员组成的高素质、多学科的小组，我们的更新计划由独特和合作的研究团队组成，他们能够加速获得对新生儿贫血和血小板减少领域至关重要的知识。在过去的17年里，我们富有成效的PPG团队(200种出版物)对新生儿输血医学和血液学的临床实践和研究产生了重大影响。我们的计划拥抱并体现了协同P01方法，这种方法已经并将使我们能够取得比我们任何人单独取得的更快的进展。通过这次更新，我们准备继续挑战范式和推进知识，同时我们在先前成果的基础上再接再厉，最大限度地开展合作。

The unifying theme of this PPG renewal is understanding the pathophysiology, and establishing the optimal management, of two of the most serious and frequently encountered conditions among critically ill, premature infants: anemia and thrombocytopenia. Current treatment for these conditions relies largely on transfusion of red blood cells and platelets¿therapies that remain controversial and incompletely investigated. Prior research by our PPG group and others has established that many aspects of the pathophysiology of the anemia and thrombocytopenia of prematurity are unique. However, our understanding is incomplete. Hence, the goals of our renewal are to further define the mechanisms that give rise to these pressing and related clinical conditions, to optimize their treatment per such mechanistic knowledge, and consequently, to reduce both short- and long-term disabilities associated with these conditions. Our 4 interrelated projects, whose objectives are directly relevant to our unifying theme, include: Project 1 "Optimized erythropoietin treatment of neonatal anemia," PL JA Widness (and P Veng-Pedersen); Project 2 "Pathogenesis and treatment of neonatal thrombocytopenia," PL M Sola-Visner; Project 3 "Preterm transfusions: brain structure/function outcomes," PL PC Nopoulos; and Project 4 "The role of neonatal anemia In learning and memory," PL MK Georgieff. Core A ("Administrative, statistical, and research personnel") and Core B ("Laboratory and database," PL DM Mock) will provide support for all projects. To enhance effectiveness, our program has expanded to involve four (vs. two) performance sites, so that data can be shared among Projects 1, 2 and 3 (i.e. data from the same infants will be used In studies at multiple sites). Our history of success conducting multisite research will be invaluable in coordinating studies across the various performance sites. Because our Program rests on a highly-qualified, multidisciplinary group of new and experienced investigators, our renewal program consists of unique and collaborative research teams capable of accelerating the acquisition of knowledge vital to the fields of neonatal anemia and thrombocytopenia. Over the past 17 years, our productive PPG group (200 publications) has made a substantial impact on clinical practice and research in neonatal transfusion medicine and hematology. Our program embraces and exemplifies the synergistic P01 approach, which has and will enable us to make more rapid progress than any of us could individually. With this renewal, we are poised to continue to challenge paradigms and advance knowledge, as we build on prior results and maximize collaboration.