Children's Research Institute Hematology Research Training Grant

描述（由申请人提供）：在儿科输血医学和血液学领域进行高质量和变革性研究的过程因以下几个障碍而变得复杂：1)缺乏强大的研究培训和实施基础设施；2)缺少中高级研究人员作为研究导师；3)需要多学科的转化方法来回答关键的临床问题；4)临床和转化研究人员在少数群体地位和性别方面缺乏多样性，尽管有不成比例的代表性不足的婴儿和少数群体儿童患有疾病和障碍，这些疾病和障碍可以从这种调查中受益。我们使用这个应用程序提出了一个高度协作的、多学科的、跨机构的指导培训项目，为未来的儿科输血医学和血液学科学家提供指导培训，以解决这些障碍。它将专注于推进血液学、心血管和肺部疾病、输血和移植的诊断和临床管理方面的临床和转化奖学金。该项目名为“儿科血液学和输血医学多学科研究培训奖”（PHTMMRT），其长期目标是培养来自不同背景的受训者，使其具备学术领导能力和独立资助的研究事业。该计划的每位学员将在乔治华盛顿大学医学与健康科学学院（GWU）完成临床与转化科学硕士学位（MsCTR），并将在多学科指导团队的联合监督下设计，实施和完成一个研究项目。在三年项目结束时，每位学员将至少在一次国家研究会议上展示他/她的数据，起草并提交一份或多份适合外部同行评审出版物的手稿，并向NIH或同等资金来源提交外部资助申请。该项目将由两位具有独特和互补的临床背景和研究专业知识的高素质美国国立卫生研究院资助的首席研究员/项目主任（pi / pd）监督。他们将得到国家儿童医院及其附属的GWU医学与健康科学学院（GWU）的9名教师和9名外部导师的支持，这些导师是凝血、输血医学、移植、镰状细胞病和血管生物学方面的研究科学家。一名多元化促进官员，一名全职教授和资助调查员，将协助招募少数民族、残疾人或弱势群体申请人和妇女。最后，由6位nih资助的输血医学和血液学领域的国家领导人组成的外部咨询委员会，将为PHTMMRT项目的发展和受训者的进步提供年度反馈和指导。PHTMMRT项目将利用国家儿童医院（CNMC）的多种现有优势，与邻近的研究机构（包括NIH、美国红十字会、FDA和霍华德大学医学院）合作，直接解决上述每个障碍，这些研究机构提供了出色的培训机会。这些优势包括：1。A)从机械调查到卫生服务研究的所有领域的转化和临床科学家的可用性；1.b)一群经验丰富的NIH资助教师，他们与我们的外部导师一起提供无与伦比的核心设施和研究培训；2)由K学者和其他学员组成的大型本地社区，通过学员委员会，为团队科学提供学院式、正式和非正式的跨学科学习机会；3)我们强大的ctsa资助基础设施，用于支持实习生研究，包括临床和转化研究的硕士学位；4)我们长期致力于招募和培训来自不同临床学科的研究人员，这些研究人员是少数民族或女性。我们的多学科、跨机构导师将培养四名未来儿科血液学和输血医学的学术领袖，他们将领导临床和转化研究团队，成功竞争资助支持，并为影响全国儿童的儿科疾病的病因、病理生理学和治疗提供新的见解。

DESCRIPTION (provided by applicant): The conduct of high quality and transformative research in pediatric transfusion medicine and hematology is complicated by several barriers including, 1) lack of a robust infrastructure for research training and implementation; 2) shortage of mid-career and senior investigators to serve as research mentors; 3) need for multidisciplinary translational approaches to answer critical clinical questions; 4) lack of diversity among clinical and translational investigators with respect to minority status and gender despite a disproportionate number of under-represented infants and children minority with diseases and disorders which could benefit from such investigations. We use this application to propose a highly collaborative and multidisciplinary, inter-institutional program of mentored training for future scientists in pediatric transfusion medicine and hematology that will address these barriers. It will focus on advancing clinical and translational scholarship on the diagnosis and clinical management of hematological, cardiovascular and pulmonary disorders, transfusion and transplantation. The program, the Pediatric Hematology and Transfusion Medicine Multidisciplinary Research Training Award (PHTMMRT) will have the long-term objective of preparing trainees from diverse backgrounds for academic leadership and independently funded research careers. Each trainee in the program will complete a Master's Degree in Clinical and Translational Science (MsCTR) at the George Washington University School of Medicine and Health Sciences (GWU) and will devise, implement and complete a research project under the joint oversight of multidisciplinary mentorship team. At the completion of the three year program, each trainee will have presented his/her data at least at one national research meeting, drafted and submitted one or more manuscripts suitable for external peer-reviewed publications, and submitted an external grant application to the NIH or equivalent funding source. The program will be overseen by two highly qualified and NIH-funded Principal Investigators/Program Directors (PIs/PDs) with distinct and complementary clinical backgrounds and research expertise. They will be supported by 9 faculty of Children's National and its affiliated GWU School of Medicine and Health Sciences (GWU) with 9 external mentors who are research scientists with expertise in coagulation, transfusion medicine, transplantation, sickle cell disease and vascular biology. A Diversity Enhancement Officer, a full professor and funded investigator, will assist in the recruitment of minority, disabled or disadvantaged applicants, and women. Finally, an External Advisory Committee, composed of 6 NIH-funded national leaders in transfusion medicine and hematology with established career mentorship programs, will offer annual feedback and guidance with respect to development of the PHTMMRT program and to the trainees' progress. The PHTMMRT program will directly address each of the barriers noted above by leveraging several of Children's National's (CNMC) multiple and pre-existing strengths with collaborating neighboring research institutions including the NIH, American Red Cross, FDA and Howard University School of Medicine which provide outstanding training opportunities. These strengths include 1.a) the availability of translational and clinical scientists across the full spectrum from mechanistic investigations to health services research; 1.b) a pool of experienced NIH funded faculty who with our external mentors provide access to unparalleled core facilities and research training; 2) our large, local community of K Scholars and other trainees which, through a Council of Trainees, will provide collegial, formal and informal, interdisciplinary learning opportunities in team science; 3) our robust CTSA-funded infrastructure for supporting trainee research, including a MS in Clinical and Translational Research; 4) our longstanding commitment to recruit and train investigators from diverse clinical disciplines who are under-represented minorities or women. Our multidisciplinary, inter-institutional mentors will train four future academic leaders in Pediatric Hematology and Transfusion Medicine, who will lead both clinical and translational research teams, compete successfully for grant support and provide novel insights into the causes, pathophysiology and treatment of pediatric diseases that affect the Nation's children.