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Blood and Marrow Transplantation
Research
The programs in Pediatric Hematology and Pediatric BMT have a rich history of productive research, both clinical and laboratory based. In the past 20 years, the major research focus in Pediatric Hematology had been on sickle cell disease. Major initiatives included a program for the prenatal diagnosis of sickle cell disease, the study of neurovascular complications in sickle cell disease, participation in the various projects of the Comprehensive Sickle Cell Network such as the Stroke Prevention Trial (STOP), the Pre-operative Transfusion study, the Hip Coring study and the Parvovirus study.
In the past 5 years, there has been an additional focus in Pediatric Hematology on iron overload disorders. The Superconducting Quantum Interference Device (SQUID), one of just 2 in the US and 4 in the world, was set up in 1999. A new, high Tc instrument has been fully operational as of 2004. Basic and clinical programs to study cardiac disease in patients with transfusional iron overload have included the study of cardiac dysfunction by 2- and 3-dimensional echocardiography and Holter monitoring as well as novel studies of structure and function of the heart in iron loaded individuals by MRI. This program is headed by Dr. Gary Brittenham and is done in collaboration with the Hatch Research MRI center at Columbia.
In the last few years there has been a significant increase in specialized research in other areas of pediatric hematology and new areas in pediatric BMT including: stem cell biology, phagocytic physiology, megakaryopoiesis, cytokine biology, natural killer cell biology, dendritic cell biology, T-cell physiology, regulatory T-Cell biology, hematopoiesis, bone marrow failure disorders, leukemia and lymphoma genomics, among others.
Basic Research
Hematology and Blood and Marrow Transplantation
Stem cell biology and blood cell development- Transplantation biology and tolerance
- Tumor immunology
- Cancer vaccines
- Natural killer cell biology and physiology
- T-cell biology and physiology
- Dendritic cell biology and physiology
- Regulation of immune cell activation and function
- Targeted immunotherapy
- Molecular genetics of blood immune cells
- Molecular genetics of childhood leukemias and lymphomas
- Imaging and quantification of iron organ deposition
- Angiogenesis (blood vessel development)
Clinical Research
Blood and Marrow Transplantation
Reduced intensity conditioning, allogeneic stem cell transplantation- Malignant Diseases Acute myeloid leukemia
- Chronic myeloid leukemia
- Hodgkin's disease
- Non-Hodgkin's lymphoma
- Neuroblastoma
- Selected solid tumors
- Sickle cell anemia
- Thalassemia
- Aplastic anemia
- Fanconi anemia
- Severe combined immunodeficiency syndrome
- Wiscott-Aldrich syndrome
- Chronic granulomatous disease
- Hurler's syndrome
- Metachromatic leukodystrophy
- Adrenoleukodystrophy
- Krabbe's disease
- Juvenile rheumatoid arthritis
- Systemic lupus erythematosus
- Scleroderma
- Rheumatoid arthritis
- Selected others
- Acute lymphoblastic leukemia
- Acute myeloid leukemia
- Chronic myeloid leukemia
- Myelodysplastic syndrome
- Neuroblastoma
- Lymphoma
- Sickle cell anemia
- Thalassemia
- Severe aplastic anemia
- Hemophagocytic lymphohistiocytosis
- Selected others
Myeloablative (full intensity) autologous stem cell transplantation
- Neuroblastoma
- Wilms' tumor
- Ewing's sarcoma
- Osteogenic sarcoma
- Germ cell tumors
- Desmoplastic round cell tumor
- Primitive neuroectodermal tumor
- Medulloblastoma
- Astrocytoma
- Ependymoma
- Glioblastoma multiforme
- Selected others
Acute graft-versus-host-disease prevention and treatment
- Mycophenolate mofetil and tacrolimus prophylaxis
- Rabbit antithymocyte globulin prophylaxis
- Alemtuzumab prophylaxis
- CD34 Stem cell selection
- T-Cell depletion
- Intra-arterial steroid and cyclosporin treatment
- Denileukin diftitox
- Mycophenolate mofetil drug metabolism
- Antithymocyte globulin drug metabolism
Stem and immune cell engraftment and reconstruction
- Donor/recipient chimerism (percent of each)
- Neutrophil cell recovery
- Platelet cell recovery
- Red blood cell recovery
- T-cell recovery
- B-cell recovery
- Dendritic cell recovery
Changes in minimal residual cancer disease
- Flow cytometry monitors
- Cytogenetic monitors
- Molecular genetic monitors
Reduction in infectious disease complications
- Gancyclovir/foscarnet CMV disease prophylaxis
- Liposomal amphotericin B prophylaxis
- Early detection of adenovirus infections
Developmental Oncology Therapeutics
New drug investigations
- Radioactive 90Y-antiCD20 for lymphomas
- Decitabine for childhood leukemias
- Decitabine doxorubicin and cyclophosphamide for solid tumors
- Depsipeptide for childhood solid tumors
- BCL-2 antisense for childhood solid tumors
- EGFR tyrosine kinase inhibitor OSI-774 for solid tumors
- Anti-angiogenic bevacizumab for solid tumors
- Topotecan, ifosfamide, carboplatin (TIC) for solid tumors
- Epidermal growth factor receptor tyrosine kinase inhibitor, ZD1839 (IressaTM) for solid tumors
- Pemetrexed for solid tumors
- Temozolomide for leukemia
- Bortezomib for leukemia
- 17-AAG for solid tumors or leukemia
- Campath-1H for acute lymphoblastic leukemia
- Epratuzamab for acute lymphocytic leukemia
Developmental Hematology Therapeutics
- ICL-670 chelator in iron overload
- NHLBI STOP I and II
- NHLBI Thalassemia Network
- NESP in sickle cell disease
- SWITCH in sickle cell disease tria
- Stem cell transplantation for sickle cell disease and thalassemia
Contact
- Pediatric Blood and Marrow Transplantation
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