Bone Marrow Transplant

Bone marrow transplants (BMT) can be a lifesaving treatment for children who have cancer, such as leukemia and lymphoma as well as diseases of the immune system, aplastic anemia that occurs when bone marrow stops producing new blood cells, inherited diseases of the bone marrow such as sickle cell anemia and some metabolic diseases.

Transplants involve replacing diseased marrow with healthy marrow, injected into the bloodstream via an intravenous tube. The marrow may come from a healthy donor or healthy stem cells may be collected from the blood of the patient.

At UCSF Children's Hospital

More than 600 transplants have been performed for children at UCSF Children's Hospital since the Pediatric Blood and Marrow Transplant Program was established in 1982. That year, we performed the first partially matched bone marrow transplant on the West Coast using bone marrow from a parent for a child with a severe immunodeficiency disease.

The program also performed the first T-cell depleted transplant on the West Coast for a child with leukemia in 1985 and more recently the first pure blood stem cell transplant from a parent to a child with severe combined immunodeficiency disease (SCID) in North America. Today, we are a leader in special treatment options for children with primary immunodeficiency diseases, marrow failure syndromes, genetic diseases, cancers and other life-threatening illnesses.

Take an online tour of the bone marrow transplant unit and clinic at UCSF Children's Hospital.

Goal of Transplant

The goal of the transplant is to replace unhealthy or destroyed bone marrow stem cells with normal bone marrow stem cells from a donor. The transplant is performed following a conditioning regimen that includes high doses of chemotherapy and sometimes radiation. The long-term survival rate varies with disease from 30 percent to 70 percent for children with leukemia to 80 percent to 95 percent for children with genetic diseases.

The BMT team at UCSF Children's Hospital is a leader in using alternative donors when a sibling with the same tissue type is not available. Alternative donors include matched unrelated volunteers or partially matched relatives.

Conditions Treated

We specialize in stem cell transplants for children with:

• Severe genetic defects such as metabolic diseases and primary immunodeficiency disorders
• Marrow failure and pre-malignant disorders such as aplastic anemia and myelodysplastic syndrome
• Life-threatening cancers such as neuroblastoma and leukemia
• Severe combined immunodeficiency disease (SCID)
• Solid tumors and brain tumor

Donor Options

When a matched related or unrelated donor cannot be found, a stem cell transplant can be done using a partially matched relative. Stem cell transplants involve collecting bone marrow cells from the blood of a parent, sibling or other close relative, and selecting the healthy stem cells for transplant.

Complete Care

Our goal is to provide the best care available in an environment that is comfortable and supportive for your child and family. We provide comprehensive clinical, nutritional and developmental evaluations as well as psychosocial support for patients and their families.

More Information

To learn more about BMT at UCSF Children's Hospital, please visit the following sections:

• Bone Marrow Transplant Process
• Bone Marrow Donors
• Specific Treatment Options
• Patient Education Materials
• Parent Handbook

For more information or to make an appointment, please call the Pediatric Blood and Marrow Transplant Program:

For help finding a bone marrow transplant specialist for children or adults, please contact our Physician Referral Service:

Reviewed by health care specialists at UCSF Children's Hospital.
Last updated May 6, 2008