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The physicians at UPMC Children’s Hospital of Pittsburgh's Blood and Marrow Transplantation and Cellular Therapies (BMTCT) program continuously strive to improve cellular therapies for treatment of childhood cancer and blood diseases.
The division’s research is pursuing new treatment methods, such as reduced-intensity conditioning, that will make marrow and cord blood transplants kinder, gentler, and safer for pediatric patients. The team’s ultimate goals are to reduce the complication rate and improve the quality of life for children undergoing these specialized procedures.
In pursuit of these goals, investigators are active on multiple fronts, using both basic laboratory research and clinical trials. In the laboratory, scientists are studying immune reconstitution, tolerance and alloreacivity, and cellular immunotherapy. Clinical investigations are underway to trial novel therapies for treating Crohn’s disease and other autoimmune diseases, Krabbe disease and other leukodystrophies, MPS syndromes, and high-risk leukemias.
Other BMTCT studies are focused on understanding the relationship between individual genetic variability and post-transplant outcomes, including graft-versus-host disease (GVHD). Our investigators have learned that individual variation in immune-response genes influences a child’s outcome after transplantation. This has led to further clinical research focused on therapies that move away from the "one size fits all" approach to tailored treatments that are based on a each patient’s specific metabolic needs.
Study of Myeloablative and Reduced-Intensity Conditioning Regimens for Children With Acute Myeloid Leukemia or Myelodysplastic Syndrome Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Autologous Stem Cell Transplantation with CD34 Selected Peripheral Blood Stem Cells (PBSC) in Pediatric and Young Adult Patients with Severe Crohn's Disease
Bilateral Orthotopic Lung Transplant (BOLT) in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant (BMT) From Partially HLA-Matched Cadaveric Donors
Study of Intravenous Gene Transfer with an AAVrh10 Vector Expressing GALC in Krabbe Subjects Receiving Hematopoietic Stem Cell Transplantation
Study of Reduced-Intensity Conditioning In Patients With Non-Malignant Disorders Undergoing Umbilical Cord, Bone Marrow, or Peripheral Blood Stem Cell Transplantation
Hematopoietic Graft Engineering: Pre-clinical Studies of Immunocompetence
T-Cell Depleted, Alternative Donor Transplant in Pediatric and Young Adult Patients with Severe Sickle Cell Disease
Use of Miltenyi Biotec’s CliniMACS CD34 Reagent System as a Humanitarian Use Device for Isolation of Hematopoietic Stem Cells or T-cell Depletion in Multiple Settings
Autologous Stem Cell Transplantation with CD34-Selected Peripheral Blood Stem Cells in Patients with Treatment-Resistant Systemic Sclerosis
Lung Transplant in Tandem with Bone Marrow Transplant for Combined Lung and Bone Marrow Failure
Protocols for a Research Database and Sample Repository for Hematopoietic Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries
Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
Trial of Naïve T Cell Depletion for Prevention of Chronic Graft-versus-host Disease in Children and Young Adults with Blood Cancers Undergoing Donor Stem Cell Transplant
Study of Letermovir in Pediatric Participants from Birth to Less than 18 Years of Age at Risk of Developing CMV Infection and/or Disease Following Allogeneic Hematopoietic Stem Cell Transplantation
Study of Unlicensed, Investigational Cryopreserved Cord Blood Units Manufactured by the National Cord Blood Program and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients
Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units for Transplantation in Pediatric and Adult Patients with Hematologic Malignancies and Other Indications
Long-term Follow-up Registry and Biorepository for Patients Diagnosed With Primary Immunodeficiencies, Malignancy, and/or Eligible to Undergo Hematopoietic Stem Cell Transplant
Pathogen Identification in Pediatric Hematopoietic Stem Cell Transplant Patients with Suspected Lower Respiratory Tract Infection
A Prospective Natural History Study of Diagnosis, Treatment, and Outcomes of Children with SCID Disorders
Xiaohua (Sophie) Chen, PhD
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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