Current Research and Scholarly Interests
Bone marrow transplantation (BMT) is a treatment modality which is being broadly applied to a growing number of disorders. Increasing success with BMT is offering improved survival to pediatric and adult patients with acute leukemia, chronic leukemia, lymphomas, and a variety of solid tumors as well as severe aplastic anemia. In the pediatric population, a variety of congenital disorders such as immunodeficiency syndromes, bone marrow failure states, hemoglobinopathies and inborn errors of metabolism can also be successfully treated with BMT. Identification of appropriate marrow donors (within or outside of the family) and the increasing use of autologous stem cells with or without purging have made this treatment approach available to more and more patients. Future studies will concentrate on control and prevention of graft-versus-host disease, ablation of malignant cells, and gene-transfer utilizing marrow stem cells as the vector.
T-cell Acute Lymphoblastic Leukemia and advanced stage Lymphoblastic Lymphoma are rare malignancies which affect pre-teen and teen-aged children with a male preponderance. These entities are closely related biologically, and probably represent different ends of a spectrum of T-cell malignancies. Treatment approaches taking advantage of the biological similarities of these entities have been successful in improving survival for affected children. Further studies of innovative approaches (immunotoxins, monoclonal antibody targeting, tumor antigen vaccination) as well as randomized clinical trials defining better chemotherapeutic regimens are ongoing. All patients entered in these protocols for treatment also participate in biologic studies utilizing molecular markers of minimal residual disease and classification studies at diagnosis and relapse including complete immunophenotyping and cytogenetics as well as classical morphology and histochemistry.