Blood and Marrow Transplantation (BMT) and Cellular Therapies

Doctors at UPMC Children's Hospital of Pittsburgh use blood and marrow transplants (BMT) to treat diseases once considered incurable, such as:

  • Certain types of cancer
  • Blood disorders
  • Immune conditions

What Is a Bone Marrow Transplant for Children?

BMT uses healthy bone marrow, or stem cells — either from the child or another donor — to replace damaged or destroyed marrow.

Types of BMT include:

  • Autologous (auto) bone marrow transplant: Your child is his or her own donor. Doctors take stem cells either from the blood or the center of a bone and filter the cells before transplanting them.
  • Allogenic (allo) bone marrow transplant: Someone who has similar genes to your child donates stem cells. This can be a relative — often a sibling — or an unrelated donor from the national bone marrow registry.
  • Cord blood transplant: Doctors take stem cells from your infant’s umbilical cord right after birth. Doctors test, type, count, and freeze the cells until needed. These stem cells mature more quickly than those taken from another donor.

CAR (Chimeric Antigen Receptor) T-Cell Therapy

UPMC Children’s Hospital of Pittsburgh is one of a small group of children's hospitals that offers CAR T-cell therapy.

We collect your child's own T-cells and send them to the lab. There, we change the T-cells so they can find and kill cancer cells.

We use the FDA-approved KYMRIAH® CAR T-cell therapy for children and young adults with relapsed or refracted B-cell acute lymphoblastic leukemia.

Why Choose UPMC Children's for Blood and Bone Marrow Transplant?

Blood and Marrow Transplant Research and Clinical Trials

Part of the mission of the Division of Blood and Marrow Transplantation and Cellular Therapies is to:

  • Design and test disease-specific BMT regimens for children with high-risk leukemia or lymphoma, and with life-threatening inherited conditions.
  • Research and evolve new cellular immune therapy programs for cancer and viral infections.
  • Find ways to make BMT safer and more effective.

The division has a strong focus on reduced-intensity conditioning (RIC)/toxicity cord blood, bone marrow, and stem cell transplant for inherited disorders.

BMT 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)

Doctors

Locations

Clinical Studies

  • Blood and Marrow Transplantation (BMT)

    UPMC Children's Hospital of Pittsburgh
    4401 Penn Avenue
    Floor 9
    Pittsburgh, PA 15224
    412-692-6740
  • 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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    Krabbe Disease Treatment with FBX-101 Gene Transfer Therapy: The RESKUE Study – Phase I/II

    Study of Intravenous Gene Transfer with an AAVrh10 Vector Expressing GALC in Krabbe Subjects Receiving Hematopoietic Stem Cell Transplantation

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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

    Read more

    Severe Combined Immunodeficiency Disorder Progression Study

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

    Read more

    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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