Blood and Marrow Transplantation (BMT) and Cellular Therapies

Our Mission

The clinical mission of the Division of Blood and Marrow Transplantation and Cellular Therapies is to design and test disease-specific and biologically rational novel reduced-toxicity transplantation regimens for patients with high-risk leukemia or lymphoma, and for those afflicted with life-threatening inherited conditions that can lead to bone marrow failure, immune deficiency, autoimmune diseases, and neurodegenerative conditions including but not limited to leukodystrophies and mucopolysaccharidosis syndromes.

Our translational research mission focuses on developing new cellular immune therapy programs for cancer and viral infections and testing the use of bone marrow transplantation following solid organ transplant to establish immunity, tolerance, and extend organ survival.

CAR T-Cell Therapy

UPMC Children’s Hospital of Pittsburgh is one of a small group of children's hospitals that offers CAR (chimeric antigen receptor) T-cell therapy, a type of immunotherapy that uses a person’s own genetically modified T cells to find and kill cancer cells. At UPMC Children’s Hospital we use KYMRIAH®, an FDA-approved CAR T-cell therapy for children and young adults with relapsed or refracted B-cell acute lymphoblastic leukemia (ALL).

Bone and Marrow Transplant Services

Blood and marrow transplantation (BMT) is used successfully to treat a number of cancers, blood diseases, and immune disorders that were once considered incurable. Our proactive patient care with close monitoring of the immune system has resulted in unsurpassed outcomes since 2010. Our one-year transplant-related mortality is below 10 percent for all allogeneic marrow, PBSC, and cord blood transplants and ~5 percent for our institutional reduced-intensity allogeneic transplant trial.

The division has a strong programmatic focus on reduced-intensity/toxicity cord blood, bone marrow, and stem cell transplantation for inherited disorders. In 2008, Dr. Szabolcs designed the first ever reduced intensity cord blood transplantation trial for inborn errors of metabolism with similar engraftment rates as ablative therapies, Biol Blood Marrow Transplant. 2014 Mar;20(3):326.

Our current, further refined Pitt trial is leading in safety and efficacy saving children/young adults from not only toxic deaths but also from brain, lung, liver, heart, GU, and other organ toxicities. The absence of severe, or extensive GVHD and improved quality of life permits early return to their local communities while our integrated Ronald McDonald House offers comfortable housing during the weeks away from home.

As an evidence of our programmatic efforts to make transplant safer and more effective, we are conducting a prospective trial for children and young adults afflicted with high-risk acute myeloid leukemia (AML) – “Reduced-Intensity Conditioning (RIC) and Myeloablative Conditioning (MAC) for HSCT in AML/MDS.”

The division has a clinical research focus on Crohn’s disease and other autoimmune disorders. Novel, reduced toxicity/intensity protocols are opening for advanced leukemia, sickle cell disease, and thalassemia.

In collaboration with other University of Pittsburgh investigators, we are the only center in the world to offer tandem lung and bone marrow transplantation for pediatric and adult patients with immunodeficiencies who have progressed to pulmonary failure. Ongoing collaborations at Pitt will pioneer further applications.

In the News

Banner Care For Our Bonus Baby
Children's blog post (9/29/14)

Teen Improving After Transplant At Children’s Hospital
CBS Pittsburgh (12/18/13)

'Yo Gabba Gabba!' trip planned for Braddock girl who calls Children's Hospital home
Pittsburgh Post-Gazette (8/29/13)



Clinical Studies

Meghan Frost, CRNP

Nicole Hogue, PA-C

Emily Mansmann, PA-C

Tammy Spight, PA-C

Rebecca Zanecosky, CRNP

  • Blood and Marrow Transplantation (BMT)

    UPMC Children's Hospital of Pittsburgh
    4401 Penn Avenue
    Floor 9
    Pittsburgh, PA 15224
  • 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 & Outcomes Review

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

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    Chronic GVHD Prevention Through Naïve T Cell Depletion in BMT Transplants for Leukemia – Phase II

    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

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    CMV Infection Prevention with Letermovir in HSCT Recipients – Phase IIb

    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

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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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    Protocol for Transplantation of Cryopreserved CBUs from the National Cord Blood Program

    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

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    Protocol for Transplantation of Cryopreserved CBUs Compliant with National Marrow Donor Program Standards

    Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units for Transplantation in Pediatric and Adult Patients with Hematologic Malignancies and Other Indications

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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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    Registry & Tissue Banking Study of Patients Undergoing HCT, SOT, or with Autoimmune Disease

    Hematopoietic Graft Engineering: Pre-clinical Studies of Immunocompetence

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    Respiratory Tract Infection Identification in HSCT Patients

    Pathogen Identification in Pediatric Hematopoietic Stem Cell Transplant Patients with Suspected Lower Respiratory Tract Infection

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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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    Sickle Cell Disease Treatment with Alternative Donor Bone Marrow Transplant

    T-Cell Depleted, Alternative Donor Transplant in Pediatric and Young Adult Patients with Severe Sickle Cell Disease

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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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    Systemic Sclerosis Treatment with Autologous Transplant of CD34-Selected Peripheral Blood Stem Cells

    Autologous Stem Cell Transplantation with CD34-Selected Peripheral Blood Stem Cells in Patients with Treatment-Resistant Systemic Sclerosis

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    Treating Bone Marrow & Lung Failure with Tandem Lung/ Marrow Transplant

    Lung Transplant in Tandem with Bone Marrow Transplant for Combined Lung and Bone Marrow Failure

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