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.

Public Health Relevance

The postdoctoral T32 training program will focus on training the next generation of pediatric clinical and translational researchers. This T32 will link trainees to physicians and basic/translational scientists whose work advances the diagnosis and management of hematological cardiovascular and pulmonary disease. This T32 will provide three years of mentored training and experience and will utilize core resources of our CTSA.

National Institute of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
Institutional National Research Service Award (T32)
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NHLBI Institutional Training Mechanism Review Committee (NITM)
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Chang, Henry
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Children's Research Institute
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Pecker, Lydia H; Kappa, Sarah; Greenfest, Adam et al. (2018) Targeted Hydroxyurea Education after an Emergency Department Visit Increases Hydroxyurea Use in Children with Sickle Cell Anemia. J Pediatr 201:221-228.e16
Pecker, Lydia H; Guerrera, Michael F; Loechelt, Brett et al. (2017) Homozygous ?-thalassemia: Challenges surrounding early identification, treatment, and cure. Pediatr Blood Cancer 64:151-155
Pecker, Lydia H; Schaefer, Beverly A; Luchtman-Jones, Lori (2017) Knowledge insufficient: the management of haemoglobin SC disease. Br J Haematol 176:515-526
Kaushal, Megha; Byrnes, Colleen; Khademian, Zarir et al. (2016) Examination of Reticulocytosis among Chronically Transfused Children with Sickle Cell Anemia. PLoS One 11:e0153244
Barriteau, Christina M; Thompson, Amanda L; Meier, Emily R et al. (2016) Sickle cell disease related internet activity is three times less frequent than cystic fibrosis related internet activity. Pediatr Blood Cancer 63:2061-2
Krivega, Ivan; Byrnes, Colleen; de Vasconcellos, Jaira F et al. (2015) Inhibition of G9a methyltransferase stimulates fetal hemoglobin production by facilitating LCR/?-globin looping. Blood 126:665-72
de Vasconcellos, Jaira F; Fasano, Ross M; Lee, Y Terry et al. (2014) LIN28A expression reduces sickling of cultured human erythrocytes. PLoS One 9:e106924
Lee, Y Terry; de Vasconcellos, Jaira F; Yuan, Joan et al. (2013) LIN28B-mediated expression of fetal hemoglobin and production of fetal-like erythrocytes from adult human erythroblasts ex vivo. Blood 122:1034-41