Children's Research Institute Hematology Research Training Grant

项目总结 在儿科输血医学和血液学方面进行高质量和变革性的研究 复杂的几个障碍包括，1)缺乏强大的研究、培训和基础设施 执行；2)缺少职业生涯中期和高级调查人员担任研究导师；3)需要 回答关键临床问题的多学科翻译方法；4)临床中缺乏多样性 以及关于少数群体地位和性别的翻译调查人员，尽管人数不成比例 代表不足的婴儿和少数族裔儿童患有可从中受益的疾病和障碍 调查。 我们使用此应用程序来建议一个高度协作、多学科、跨机构的计划 为未来的儿科输血医学和血液学科学家提供指导培训，以解决这些问题 障碍。它将专注于推进诊断和临床方面的临床和翻译学术 血液、心血管和肺部疾病的管理、输血和移植。这个 儿科血液学与输血医学多学科研究培训 Awards(PHTMMRT)将有一个长期目标，为来自不同背景的学员做好准备 学术领导力和独立资助的研究事业。计划中的每个实习生都将完成一项 乔治华盛顿大学理工学院临床与翻译科学硕士学位 (GWU)，并将设计、实施和完成一项由 联合监督多学科指导团队。在三年课程结束时，每个学员 将至少在一次国家研究会议上提交他/她的数据，起草并提交一份或多份 适合于外部同行评议出版物的手稿，并向 美国国立卫生研究院或同等资金来源。该项目将由两名非常合格的NIH资助的 首席研究人员/项目主任(PI/PD)具有截然不同和互补的临床背景和 研究专业知识。他们将得到国立儿童学院及其附属的GWU学校9名教职员工的支持 医学与健康科学(GWU)，有9名外部导师，他们是具有以下专长的研究科学家 凝血、输血医学、移植、镰状细胞病和血管生物学。A多样性 加强干事是一名全职教授和受资助的调查员，将协助招募少数族裔， 残疾或弱势申请者，以及妇女。最后，由6人组成的外部咨询委员会 NIH资助的输血医学和血液学领域的国家领导者，并建立了职业指导 计划，将就PHTMMRT计划的发展提供年度反馈和指导 为学员的进步干杯。 PHTMMRT计划将通过利用以下几个方面直接解决上述每个障碍 儿童国家(CNMC)与邻近研究合作的多重和预先存在的优势 包括NIH、美国红十字会、FDA和霍华德大学医学院在内的机构 提供出色的培训机会。这些优势包括：1)可提供翻译和 从机械调查到卫生服务研究的所有临床科学家；1.b)a 经验丰富的NIH资助的教职员工与我们的外部导师一起提供访问无与伦比的核心的途径 设施和研究培训；2)我们庞大的本地K学者和其他受训人员社区，通过 学员理事会，将在团队中提供合议式、正式和非正式的跨学科学习机会 科学；3)我们强大的CTSA资助的基础设施，用于支持学员研究，包括临床硕士学位 和翻译研究；4)我们长期致力于从不同的领域招募和培训调查人员 代表不足的少数族裔或女性的临床学科。 我们的多学科、跨机构导师将培训四位未来的儿科血液学学术领袖 和输血医学，谁将领导临床和翻译研究团队，成功竞争 为赠款提供支持，并对儿科疾病的病因、病理生理学和治疗提供新的见解 影响这个国家孩子的疾病。

PROJECT SUMMARY 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.