At the UCSF Pediatric Blood and Marrow Transplant Program, an essential part of our mission is to improve stem cell transplantation so that it can benefit even more patients. That's why we are continually investigating a variety of specific treatment options, also known as research protocols, for children in need of a bone marrow transplant (BMT). These include treatments for patients who have:

  • Abnormal or defective bone marrow stem cells
  • Cancer that may be cured with a BMT
  • A genetic or inherited metabolic disease that affects many organs

Patients participating in a research protocol are treated in a uniform manner, so that medical scientists can compare outcomes. These results, after collection and analysis, may be published in a scientific journal. In the case of a BMT study, the results are used to develop improved treatment options for children and adults who could benefit from a BMT.

Research protocols

At UCSF Benioff Children's Hospitals, we offer investigatory treatment protocols to support BMT in patients with the following conditions:

  • Leukemia, including acute lymphocytic leukemia and acute myelogenous leukemia
  • Hodgkin's disease and non-Hodgkin's lymphoma
  • Solid tumors, including brain tumors and sarcomas
  • High-risk neuroblastoma
  • Severe combined immunodeficiency (SCID) and other serious immunodeficiency disorders
  • Inherited metabolic disorders
  • Bone marrow stem cell defects
  • Inherited blood disorders, such as thalassemia and sickle cell disease

Reducing risks

Although BMT remains the best and sometimes only option to treat some diseases, it carries certain risks, including harm to healthy tissues, such as the brain, lungs, liver and kidneys. To minimize this damage, our program has developed novel protocols, which include the following: