This application describes plans at the University of Pennsylvania and Children's Hospital of Philadelphia to train the next generation of leaders in clinical and translational research in non-malignant hematology. The breadth and depth of the hematology programs, along with additional resources at the University, provide an ideal setting for this career development program, which will be structured to cover four areas including: Hemostasis, Red Cell Disorders, Immune Hematology and Transfusion Medicine, and Stem Cells and Myeloproliferative Disorders. Experienced faculty members will lead each area and aid the Director and Co- Directors in developing a comprehensive curriculum in benign hematology. These faculty will serve as Primary Mentors, and with Secondary Mentors, will train scholars in their areas of expertise. In addtion, the program will utilize the resources within the recently developed Institute for Translational Medicine and Therapeutics. The specific training goals of this program are: 1) To provide broad-based clinical training and exposure to all aspects of pediatric and adult non-malignant hematology;2) To guide the Scholar in acquiring the knowledge and tools necessary to be a successful clinical or translational researcher, including training in biostatistics, epidemiology, clinical trials design, and in the responsible conduct of research;3) To develop scholars with a thorough understanding of advances in basic molecular and cell biology relevant to their areas of clinical research focus, and in methodologies used in these areas;4) To ensure knowledge and understanding of ethical issues in both basic and clinical biomedical research;and, 5) To provide close mentoring of the scholars with defined timelines to meet goals in education, research, and scientific and grant writing, and to provide positive clinical research role models in a supportive academic environment. The program will train future clinical and translational researchers in benign hematology who are equipped with a sophisticated background in basic science and clinical research methodologies. It is anticipated that graduates of this program will be at the leading edge of their research field and ideally situated to compete for further NIH funding and to serve as faculty members in non-malignant hematology in academic centers, training the next generation of non-malignant hematologists.

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Physician Scientist Award (Program) (PSA) (K12)
Project #
5K12HL087064-05
Application #
7916483
Study Section
Special Emphasis Panel (ZHL1-CSR-Z (S1))
Program Officer
Werner, Ellen
Project Start
2006-09-01
Project End
2012-04-30
Budget Start
2010-09-01
Budget End
2012-04-30
Support Year
5
Fiscal Year
2010
Total Cost
$396,349
Indirect Cost
Name
University of Pennsylvania
Department
Internal Medicine/Medicine
Type
Schools of Medicine
DUNS #
042250712
City
Philadelphia
State
PA
Country
United States
Zip Code
19104
Babushok, Daria V; Stanley, Natasha; Xie, Hongbo M et al. (2017) Clonal Replacement Underlies Spontaneous Remission in Paroxysmal Nocturnal Haemoglobinuria. Br J Haematol 176:487-490
Betensky, Marisol; Babushok, Daria; Roth, Jacquelyn J et al. (2016) Clonal evolution and clinical significance of copy number neutral loss of heterozygosity of chromosome arm 6p in acquired aplastic anemia. Cancer Genet 209:1-10
Bhoj, Vijay G; Arhontoulis, Dimitrios; Wertheim, Gerald et al. (2016) Persistence of long-lived plasma cells and humoral immunity in individuals responding to CD19-directed CAR T-cell therapy. Blood 128:360-70
Ellebrecht, Christoph T; Bhoj, Vijay G; Nace, Arben et al. (2016) Reengineering chimeric antigen receptor T cells for targeted therapy of autoimmune disease. Science 353:179-84
Babushok, Daria V; Grignon, Anne-Laure; Li, Yimei et al. (2016) Disrupted lymphocyte homeostasis in hepatitis-associated acquired aplastic anemia is associated with short telomeres. Am J Hematol 91:243-7
Babushok, Daria V; Bessler, Monica; Olson, Timothy S (2016) Genetic predisposition to myelodysplastic syndrome and acute myeloid leukemia in children and young adults. Leuk Lymphoma 57:520-36
Babushok, Daria V; Olson, Timothy S; Bessler, Monica (2015) Somatic Mutations and Clonal Hematopoiesis in Aplastic Anemia. N Engl J Med 373:1673
Wong, Trisha E; Delaney, Meghan; Gernsheimer, Terry et al. (2015) Antithrombin concentrates use in children on extracorporeal membrane oxygenation: a retrospective cohort study. Pediatr Crit Care Med 16:264-9
Babushok, Daria V; Perdigones, Nieves; Perin, Juan C et al. (2015) Emergence of clonal hematopoiesis in the majority of patients with acquired aplastic anemia. Cancer Genet 208:115-28
Babushok, Daria V; Xie, Hongbo M; Roth, Jacquelyn J et al. (2014) Single nucleotide polymorphism array analysis of bone marrow failure patients reveals characteristic patterns of genetic changes. Br J Haematol 164:73-82

Showing the most recent 10 out of 35 publications