The UCSF Children's Hospital Blood and Marrow Transplant Program offers a variety of specific treatment options for children, including options for those who have:

• Bone marrow stem cells that are abnormal or defective
• Cancer that may be cured with a bone marrow transplant
• A genetic or inherited metabolic disease that affects many organs in the body

These treatment options, called protocols, are all considered research protocols since the patients who participate are treated in a uniform manner and the results, after being collected and evaluated, may be published in a scientific journal. The results of these research protocols are used to advance the use of bone marrow transplantation and allow doctors to develop improved treatment options for all children and adults who may benefit from this type of therapy.

Disease Protocols

At UCSF Children's Hospital, we offer treatment protocols for various diseases, including:

• Leukemia, including acute lymphocytic leukemia (ALL), acute myelogenous leukemia (AML)
• Hodgkins and non-Hodgkins lymphoma
• Solid tumors including brain tumors and sarcomas
• High-risk neuroblastoma
• Severe combined immunodeficiency disease (SCID) and other severe immunodeficiency disorders
• Genetic diseases, also called metabolic diseases
• Marrow stem cell defects
• Hemoglobinopathies

Reducing the Risk of Transplant

Although bone marrow transplant (BMT) remains the best and sometimes only treatment option for some diseases, it does carry certain risks, including damage to healthy tissues such as the brain, lungs, liver and kidneys. While this risk is rather great with a regular BMT, the UCSF Children's Hospital BMT Program has developed novel protocols to minimize the damage caused by the transplant process with the following:

• Reduced Intensity Conditioning
• Busulfan Pharmacokinetics
• Haplocompatible Transplants
• Umbilical Cord Blood Transplants

Reviewed by health care specialists at UCSF Children's Hospital.
Last updated October 15, 2009