Blood and Marrow Transplantation and Cellular Therapies Research

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.


Clinical Studies

Acute Myeloid Leukemia or Myelodysplastic Syndrome Transplant Using Reduced Intensity Conditioning – Phase II

Study of Myeloablative and Reduced-Intensity Conditioning Regimens for Children With Acute Myeloid Leukemia or Myelodysplastic Syndrome Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

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Blood and Marrow Transplant Research Repository & Database

Protocols for a Research Database and Sample Repository for Hematopoietic Cell Transplantation, Other Cellular Therapies and Marrow Toxic Injuries

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Chronic Granulomatous Disease Progression and Outcomes Review

Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995

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Crohn's Disease Treatment using Blood Stem Cells – Phase I/II

Autologous Stem Cell Transplantation with CD34 Selected Peripheral Blood Stem Cells (PBSC) in Pediatric and Young Adult Patients with Severe Crohn's Disease

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Immunodeficiency and End-Stage Lung Disease Treatment with BOLT+BMT Procedure

Bilateral Orthotopic Lung Transplant (BOLT) in Tandem With CD3+ and CD19+ Cell Depleted Bone Marrow Transplant (BMT) From Partially HLA-Matched Cadaveric Donors

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Non-Malignant Marrow Disorder Treatment using Reduced Intensity Conditioning – Phase II

Study of Reduced-Intensity Conditioning In Patients With Non-Malignant Disorders Undergoing Umbilical Cord, Bone Marrow, or Peripheral Blood Stem Cell Transplantation

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Registry & Tissue Banking Study of Cancer, Blood Disorder & Immunodeficiency Patients

Long-term Follow-up Registry and Biorepository for Patients Diagnosed With Primary Immunodeficiencies, Malignancy, and/or Eligible to Undergo Hematopoietic Stem Cell Transplant

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Severe Combined Immunodeficiency Disorder Progression Study

A Prospective Natural History Study of Diagnosis, Treatment, and Outcomes of Children with SCID Disorders

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Stem Cell Transplant with CliniMACS® Device

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

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Wiskott-Aldrich Syndrome Progression and Outcomes Review

Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990

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Additional Researchers

Xiaohua (Sophie) Chen, PhD