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.

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

Paul Szabolcs, MD

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Jessie L Barnum, MD

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Craig Alan Byersdorfer, MD, PhD

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Randy M Windreich, MD

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  • Blood and Marrow Transplantation (BMT)

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

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

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    National Marrow Donor Program

    National Marrow Donor Program Recipient Research Database and Research Sample Repository for Patients Undergoing Unrelated Marrow Transplants

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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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