Neonatal Anemia and Thrombocytopenia: Pathophysiology and Treatment

PPG更新的统一主题是了解危重早产儿中最严重和最常见的两种疾病的病理生理学，并建立最佳管理：贫血和血小板减少症。目前对这些疾病的治疗主要依赖于输注红细胞和血小板治疗，这些治疗仍然存在争议和不完全的研究。我们的PPG小组和其他人先前的研究已经确定，早产儿贫血和血小板减少症的病理生理学的许多方面是独特的。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引起这些紧迫和相关临床疾病的机制，根据这些机械知识优化治疗，从而减少与这些疾病相关的短期和长期残疾。我们的4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“新生儿血小板减少症的发病机制和治疗”，PL M Sola-Visner;项目3“早产儿输血：脑结构/功能结局”，PL PC Nopoulos;和项目4“新生儿贫血在学习和记忆中的作用”，PL MK Georgieff。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，我们的计划已经扩展到涉及四个（而不是两个）性能研究中心，以便项目1、2和3之间可以共享数据（即来自相同婴儿的数据将用于多个研究中心的研究）。我们成功进行多站点研究的历史将在协调各个性能站点的研究方面发挥宝贵作用。由于我们的计划依赖于一个高素质的，多学科的新的和经验丰富的研究人员小组，我们的更新计划包括独特的和协作的研究团队，能够加快知识的获取至关重要的新生儿贫血和血小板减少症的领域。在过去的17年里，我们富有成效的PPG团队（200篇出版物）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。我们的计划包含并验证了协同P01方法，该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并推进知识，同时在之前成果的基础上再接再厉并最大限度地扩大合作。

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.