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?

• Our one-year BMT survival rate is 94 percent. The death rate is below 10 percent for all allo, PBSC, and cord blood transplants. And it's under 5 percent for our reduced-intensity allo transplant trial.
• Our proactive care and close eye on the immune system has resulted in unmatched outcomes since 2010.
• We're the only center in the world to offer tandem lung and BMT for those with immune deficiencies and have lung failure.

Blood and Marrow Transplant Research and Clinical Trials

As a nationally recognized research center, UPMC Children’s Hospital’s Division of Blood and Marrow Transplantation participates in the latest clinical studies, which involve human volunteers and are intended to add to our medical understanding.

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.

• In 2008, Dr. Szabolcs designed the first-ever RIC cord blood transplant trial for children inborn metabolic disorders.
• We're conducting a prospective RIC trial for children and young adults who have high-risk acute myeloid leukemia.
• We have a current study on the use of auto stem cell transplant to help severe Crohn’s disease and other autoimmune disorders.
• Novel, RIC/intensity protocols are opening for advanced leukemia, sickle cell disease, and thalassemia.