Overview
For most of my career in Pediatric Hematology/Oncology I have focused on the use of stem cell transplant for the treatment of pediatric leukemias (ALL, AML, CML and JMML) and other non-malignant blood disorders, such as sickle cell disease, hemaphagocytic disorders, Wiskott-Aldrich, aplastic anemia, Diamond-Blackfan Anemia, as well as inherited metabolic diseases. In addition to focusing on determining the best use of stem cell transplants for these disorders, I have also been involved in clinical research investigating the prevention and treatment of transplant related morbidity, particularly veno-occlusive disease of the liver, infections and diffuse alveolar hemorrhage. As study chair for the Children's Oncology Group protocol 9904, I was involved in the development, implementation and analysis of a large, international frontline study of childhood acute lymphoblastic leukemia. Results from this study show that a significant number of children with certain favorable cytogenetic abnormalities in their leukemic cells and who have a rapid response to their initial chemotherapy can expect to have a >95% chance of cure when treated with relatively low intensity chemotherapy.
I have concentrated on providing high quality care for high risk leukemia patients who require high intensity therapies, such as stem cell transplant and immunotherapy. As a member of the Pediatric Transplant and Cellular Therapy Division I provide clinical care for these patients. As a member of various cooperative groups and local PI for several drug trials, I have worked to provide better care and more specific therapies for the toxicities associated with stem cell transplant.
I have also collaborated with the Pediatric Immunology Division to provide a life-saving therapy for a small group of patients with thymic dysfunction, which causes severe immunodeficiency. Our clinical team now provides support during these patients hospital admissions for donor thymus tissue implantation. We once again achieved a new record for the number of implanted patients during the 2022-2023 academic year.
I have concentrated on providing high quality care for high risk leukemia patients who require high intensity therapies, such as stem cell transplant and immunotherapy. As a member of the Pediatric Transplant and Cellular Therapy Division I provide clinical care for these patients. As a member of various cooperative groups and local PI for several drug trials, I have worked to provide better care and more specific therapies for the toxicities associated with stem cell transplant.
I have also collaborated with the Pediatric Immunology Division to provide a life-saving therapy for a small group of patients with thymic dysfunction, which causes severe immunodeficiency. Our clinical team now provides support during these patients hospital admissions for donor thymus tissue implantation. We once again achieved a new record for the number of implanted patients during the 2022-2023 academic year.
Current Appointments & Affiliations
Professor Emeritus of Pediatrics
·
2024 - Present
Pediatrics, Transplant and Cellular Therapy,
Pediatrics
Recent Publications
Newborn Screening for Hurler Syndrome Facilitates Early Transplant and Good Outcomes.
Journal Article Pediatr Neurol · February 2025 BACKGROUND: Hematopoietic cell transplantation (HCT) is the standard of care treatment for children with Hurler syndrome (HS). This study describes the impact of newborn screening (NBS) on HCT outcomes for these patients. METHODS: Retrospective study of HS ... Full text Link to item CiteSafety and feasibility of umbilical cord blood transplantation in children with neuronal ceroid lipofuscinosis: a retrospective study.
Journal Article Stem Cells Transl Med · January 17, 2025 Ceroid lipofuscinosis neuronal (CLN) encompasses rare inherited neurodegenerative disorders that present in childhood with clinical features including epilepsy, psychomotor delay, progressive vision loss, and premature death. Published experience utilizing ... Full text Link to item CitePathobiological signatures of dysbiotic lung injury in pediatric patients undergoing stem cell transplantation.
Journal Article Nat Med · July 2024 Hematopoietic cell transplantation (HCT) uses cytotoxic chemotherapy and/or radiation followed by intravenous infusion of stem cells to cure malignancies, bone marrow failure and inborn errors of immunity, hemoglobin and metabolism. Lung injury is a known ... Full text Link to item CiteRecent Grants
Hematology & Transfusion Medicine (T32)
Inst. Training Prgm or CMEPreceptor · Awarded by National Institutes of Health · 1975 - 2026PHASE 3 STUDY EVALUATING GENE THERAPY BY TRANSPLANTATION OF AUTOLOGOUS CD34+ STEM CELLS TRANSDUCED EX VIVO WITH THE LENTIGLOBIN BB305 LENTIVIRAL VECTOR IN SUBJECTS WITH SEVERE SICKLE CELL DISEASE
Clinical TrialPrincipal Investigator · Awarded by Bluebird Bio · 2020 - 2026A Phase III Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation with Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients with Severe Aplastic Anemia (TransIT)
Clinical TrialPrincipal Investigator · Awarded by National Marrow Donor Program · 2023 - 2025View All Grants
Education, Training & Certifications
Washington University in St. Louis ·
1987
M.D.
Washington University in St. Louis ·
1987
Ph.D.