Blood and Marrow Transplantation
As one of the world's largest, most respected pediatric services, the Blood and Marrow Transplantation (BMT) Program attracts patients from around the globe.
A section within the Children's Center for Cancer and Blood Diseases, BMT is known as a friendly environment for children, treating patients with all forms of childhood diseases treatable by hematopoietic stem cell transplantation.
At CHLA, we specialize in treating children of all ages, especially very young patients. More than half of all transplants we complete are for children under the age of five and we perform both Autologous and Allogenic Transplants.
Through our Transfusion Medicine program we offer stem cell collection, granulocyte collection, blood collection, and therapeutic apheresis procedures, with a staff with over 80 years of cumulative experience in pediatric apheresis.
BMT maintains annual certification by the Foundation for the Accreditation of Cellular Therapy (FACT). FACT accreditation has become a major index of the high quality programs for Blood and Marrow Transplantation. The certificate process includes reviews of our clinical care, stem cell laboratory, and clinical data management and outcomes assessment components of our program.
Children's Hospital Los Angeles has performed more than 1,505 bone marrow transplants since 1983, completing 70 to 75 procedures annually. Our HSCT Program is one of the largest, most highly regarded pediatric programs in the world and responsible for 75 percent of all pediatric bone marrow transplants in Los Angeles County.
- Advancing Care
- Conditions Treated
- SCID Treatment
Our team of physicians, nurses and laboratory scientists is renowned for its many innovative research and clinical advances. Our hospital is the operating center for the multi-national “Head Start” consortium and the national New Approaches to Neuroblastoma Therapy (NANT) consortium with of which include Autologous Blood and Marrow Transplant therapies.
Our institution is one of the primary referral centers in the U.S. for children born with severe combined immune deficiency (SCID).
- In 1993, Children's Hospital Los Angeles physicians were the first to use gene therapy for newborns with SCID and they continue to remain at the forefront of this emerging area.
- Doctors at our hospital have developed innovative, new clinical approach for treating children with SCID, with the goal of restoring all aspects of protective immunity with fewer side-effects than standard transplant approaches.
We offer solutions to patients with advance-stage and high-risk cancers from whom there may be no other viable treatment.
We perform allogeneic transplants, using stem cells from related or unrelated donors, peripheral blood stem cells or cord blood, providing care for patients with advanced-stage and high-risk diseases, including:
Anemia and Marrow Failure
- Aplastic anemia
- Fanconi’s anemia
- Other marrow failures
Inherited Blood Diseases
- Chronic Granulomatous disease
- Hereditary Lymphohistiocytosis
- Hurler’s syndrome
- Severe Combined Immune Deficiency
- Sickle Cell disease
- Thalassemia syndromes
- Wiskott-Aldrich syndrome
- X-linked adrenoleukodystrophy
- X-linked Hyper IgM syndrome
- Brain tumors
- Choroid Plexus Carcinoma
- Germ cell tumor
- Glioma (malignant)
- Desmoplastic Small Round Cell Tumor
- Ewing Sarcoma
- Rhabdoid Tumor of Kidney or Liver
- Rhabdomyosarcoma (recurrent)
- Retinoblastoma (high-risk)
- Wilm's Tumor (recurrent)
Our BMT Program offers a 14-bed nursing unit with HEPA-filtered isolation rooms, specifically suited for the care of pediatric patients underging hematopoeitic stem cell transplantation.
Patients are attended to by nurses who staff to a high ratio per patient and two full-time social workers.
Patient rooms feature advanced airflow, designed to protect patients from airborne germs. In addition, two negative pressure rooms are available for children who have infections that could spread to other patients.
An on site infusion center provides blood transfusions for children as outpatients once they are ready for discharge from the hospital.
Patients with Severe Combined Immunodeficiency Syndrome (SCIDS) can only be cured by a successful transplantation of hematopoietic stem cells from a healthy donor.
For SCID infants who have a brother or sister who is a "match", the transplant process is relatively easy, with a high rate of success. However, most infants with SCIDS do not have a matched family donor and therefore need a hematopoietic stem cell transplant from another donor.
For many years, the best treatment option for infants with SCID who needed a hematopoietic stem cell transplant but did not have a matched sibling was to use the bone marrow from one of the parents.
However, parents only half-match their children and therefore these transplants may be complicated by immune reactions against the child’s body (graft-versus-host disease). More recently, the National Marrow Donor Program was established as a registry of more than 10 million potential donors.
The use of hematopoietic stem cells from unrelated adult bone marrow donors or from banked umbilical cord blood has emerged as a way to find better matched stem cells. Additionally, new combinations of medicines have been identified to facilitate the engraftment of the donor stem cells with less side-effects than previous approaches could have. Based on these advances, we have developed a new clinical research protocol for the treatment of infants with SCID who lack a matched sibling donor.