The primary objective of the Pediatric Hematology Research Training Program, which is entering its 40th year of funding, has been to prepare individuals ? mostly pediatric hematology/oncology fellows ? interested in pediatric benign hematology for careers in academic medicine and biomedical research. We believe that this is a highly underserved area of research, that our Division has excelled in providing such training, and that this T32 has over the years been key to that success. This post-doctors only T32 has 4 slots per year. Of the 30 trainees on this T32, 21 are in academic faculty positions ? mostly in pediatric benign hematology, 3 in senior private industry/ government positions and 3 are current trainees.
Specific aims of this T32 include (1) the provision of research training opportunities in a wide variety of basic and clinical research areas that are of keen interest to present day research interests including studies of the hemoglobinopathies and other anemias, hemostasis and thrombosis, megakaryocytes and platelet development, immunohematology, stem cell development and bone marrow failure with a focus on translating these efforts by regenerative medicine technologies, cellular therapeutics and gene therapy to patient care; (2) the opportunity for structured coursework in bioinformatics, cell and molecular biology, immunology, biochemistry, gene transfer, biostatistics, epidemiology and research design; (3) careful monitoring of the progress of individual trainees, the overall success of the training program, and working towards individuals reaching the appropriate next academic milestones using a novel ?step-wise academic monitoring? to carry the fellows from their clinical years to the early assistant professor years; and (4) an emphasis on skills and accomplishments critical for successful academic careers such as preparation of manuscripts, presentations at seminars and national meetings, submission of grant applications, detailed bioethical training and training in mentoring. The training faculty consists of a closely-knit group of physicians and scientists who have strong records as mentors and productive investigators, who have competed successfully for NIH and other federal funding, and who have largely interacted for many years in training and research activities. Additional trainers include others on the Children's Hospital and University of Pennsylvania combined campus that would bring in key skills to a mentoring team that we believe are important for our trainees to acquire and by the recruitment of new, outstanding faculty members, and establishing contacts with industry that can provide insights into how to translate research into a clinical reality. The program has been successful in the research training of minority students at all levels to increase the numbers of these individuals in biomedical careers. Thus, we believe that despite a national trend away from pediatric subspecialty research training and toward clinical practice, our Program has continued to attract outstanding candidates and to prepare them through this T32 mechanism for careers as biomedical investigators in academic institutions, and national and international leaders in many areas of benign hematology. We believe that this renewal will show the continued evolution of our training program to meet present-day challenges in our field and provide excellence in training.

Public Health Relevance

Researchers in pediatric hematology play an important role in the diagnosis and treatment of common inherited disorders including sickle cell disease, thalassemia, hemophilia and bone marrow failure. Better treatments for these and other blood diseases seen in children will improve the quality of life of affected individuals and will reduce admissions to children's hospitals, where 50% of all admissions are for inherited diseases such as these. This proposed training program for young pediatric scientist will ensure that we have the very best people to continue to advance our understand of these diseases and come forward with ways to treat and cure these disorders.

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Institutional National Research Service Award (T32)
Project #
5T32HL007150-44
Application #
9966020
Study Section
NHLBI Institutional Training Mechanism Review Committee (NITM)
Program Officer
Mondoro, Traci
Project Start
1976-07-01
Project End
2022-06-30
Budget Start
2020-07-01
Budget End
2021-06-30
Support Year
44
Fiscal Year
2020
Total Cost
Indirect Cost
Name
Children's Hospital of Philadelphia
Department
Type
DUNS #
073757627
City
Philadelphia
State
PA
Country
United States
Zip Code
19146
Gollomp, Kandace; Kim, Minna; Johnston, Ian et al. (2018) Neutrophil accumulation and NET release contribute to thrombosis in HIT. JCI Insight 3:
Greineder, Colin F; Johnston, Ian H; Villa, Carlos H et al. (2017) ICAM-1-targeted thrombomodulin mitigates tissue factor-driven inflammatory thrombosis in a human endothelialized microfluidic model. Blood Adv 1:1452-1465
George, Lindsey A; Sullivan, Spencer K; Giermasz, Adam et al. (2017) Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. N Engl J Med 377:2215-2227
Russell, Stephen; Bennett, Jean; Wellman, Jennifer A et al. (2017) Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet 390:849-860
Oved, Joseph H; Lee, Christina S Y; Bussel, James B (2017) Treatment of Children with Persistent and Chronic Idiopathic Thrombocytopenic Purpura: 4 Infusions of Rituximab and Three 4-Day Cycles of Dexamethasone. J Pediatr 191:225-231
Arruda, Valder R; Doshi, Bhavya S; Samelson-Jones, Benjamin J (2017) Novel approaches to hemophilia therapy: successes and challenges. Blood 130:2251-2256
Bdeir, Khalil; Gollomp, Kandace; Stasiak, Marta et al. (2017) Platelet-Specific Chemokines Contribute to the Pathogenesis of Acute Lung Injury. Am J Respir Cell Mol Biol 56:261-270
Gollomp, Kandace; Lambert, Michele P; Poncz, Mortimer (2017) Current status of blood 'pharming': megakaryoctye transfusions as a source of platelets. Curr Opin Hematol 24:565-571
Nguyen, G N; George, L A; Siner, J I et al. (2017) Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A. J Thromb Haemost 15:110-121
George, Lindsey A (2017) Hemophilia gene therapy comes of age. Blood Adv 1:2591-2599

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