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Scientific Committee on Blood Disorders in Childhood

Committee Roster

Chair
Nicole Kucine, MD, MS  ('24)

Vice Chair
Julie  A. Jaffray, MD  ('24)

Appointed Members
Lionel Blanc, PhD  ('24)
Giuliana Ferrari, PhD  ('27)
Peter  M. Gordon, MD  ('24)
Kelly  Marie Knee, PhD  ('26)
Alisa  B. Lee-Sherick, MD  ('24)
Tamara  P. Miller, MD  ('24)
Sarah  K. Tasian, MD  ('24)
Ngoc Tung Tran, PhD  ('26)

Staff Liaison
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Committee Mandate

The Scientific Committee on Blood Disorders in Childhood is focused on all issues related to pediatric hematology. This includes diseases that occur exclusively in children including those that extend from the neonatal period through childhood to adult life. It is important to note that although some of these diseases may be rare, expertise in diagnosis and long-term management lies within the pediatric hematology community.

Areas of biological/mechanistic interest:
Childhood diseases are frequently inherited and often originate during embryogenesis. Moreover, many childhood blood diseases perturb normal tissue development, offering unique windows into these pathways. For these reasons, our Committee places a strong emphasis on genetics and developmental biology as they apply to aberrant immune, hematologic and malignant diseases. Specific examples include the Developmental Biology of Hematopoiesis (e.g. Transcriptional Regulation of Erythropoiesis, Ontology of Megakaryopoiesis and Regulation of White Cell Development), Developmental Hemostasis, Immune System Development, and Genetic Disorders.

The clinical areas:
Malignant and non-malignant pediatric diseases include but are not limited to:

1. Congenital disorders of the hematopoietic system (bone marrow failure syndromes, congenital neutropenias, congenital neutrophil and platelet function defects)
2. Congenital red cell disorders (e.g. hemoglobinopathies, Diamond Blackfan anemia)
3. Immune dysregulation/dysfunction (e.g. congenital and acquired HLH) and primary immunodeficiencies
4. Inflammatory diseases of childhood
5. Malignant disorders of infancy (e.g. infant ALL, JMML) and childhood (e.g. more common leukemias and lymphomas)
6. Cancer predisposition syndromes (e.g. LiFraumeni and others)
7. Congential coagulation disorders (e.g. hemophilia)
8. Neonatal hematology (e.g. maternally aquired cytopenias, intrinsic defects in hematopoiesis)

The methodological aspects:

The committee focuses on all aspects of research spanning basic sciences, translational research and clinical research. This includes turning genetic and immunological discoveries into therapy for malignant and non-malignant diseases of childhood. Specific examples are:

1. Using the Genetics of the Autoimmune Lymphoproliferative Syndrome to Guide Therapy
2. Using Leukemia Gene Rearrangements for risk adapted therapies
3. Developing Treatments for epigenetic diseases through manipulation of chromatin modifiers.
4. Genetic Characterization of Hemophilia and other single gene defects of the hematopoietic system and the Implications for Novel Therapies
5. EBV-specific T-cell therapies for EBV-associated lymphoproliferation

The emerging areas: 

1. New therapies for cancer treatment such as immune-based therapies, small molecules and risk adapted therapies to minimize late effects and to target aberrant microenvironments associated with malignant and non-malignant disease
2. New therapies for congenital, non malignant disorders including advances in stem cell transplantation and gene therapy
3. Genomics/proteomic approaches to guide diagnosis, prognosis and treatment of pediatric hematologic diseases.
4. Use of stem cell therapies to model and treat disorders (ES and iPS cells) as well as to treat complications associated with hematopoietic stem cell transplantation including graft-failure, infection, relapse, and graft-versus-host disease (MSCs, MAPCs, and T-cells).

Potential overlap and resolution with other scientific committees:

Our "age-oriented" committee has occasional overlap with other '"topic-oriented" committees (for example the ad hoc committee on marrow failure, but there is no systematic overlap that needs to be addressed.

Thus, appropriate sessions at the annual meeting may include: 

Appropriate sessions at the annual meeting may include any presentation that addresses hematological diseases that occur in childhood. Topics will cover mechanisms of disease pathophysiology, associated normal developmental processes and innovative approaches to diagnosis and treatment. The committee strives to represent both benign and malignant diseases in each program.

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