Blood and marrow transplants are most frequently used to treat children who have cancers or blood disorders. They also can be used to help children born with congenital immunodeficiency syndromes or metabolic diseases.

Hematopoietic Stem cells are the “mother cells” from which all other blood cells originate. In stem cell transplantation we use these cells to produce new generations of blood cells and immune cells.

Treatable Diseases

These diseases can be treated by blood or marrow stem transplants:

Malignant Disorders

  • Leukemias
  • Lymphomas
  • Brain tumors
  • Bone tumors
  • Soft tissue tumors

Hematologic Disorders and Bone Marrow Failure Syndromes

  • Sickle cell disease
  • Thalassemia
  • Severe aplastic anemia
  • Fanconi anemia
  • Diamond-Blackfan anemia
  • Sideroblastic anemia

Immunodeficiency Syndromes

  • Severe combined immunodeficiency (SCID)
  • Wiskott-Aldrich Syndrome
  • Cartilage hair hypoplasia
  • Chronic granulomatous disease
  • Leukocyte adhesion defects

Inborn Errors of Metabolism

  • Hurler’s Syndrome
  • Hunter’s Syndrome
  • Adrenoleukodystrophy
  • Krabbe’s disease
  • Maroteaux-Lamy syndrome
  • Osteopetrosis

Categories of Blood and Marrow Transplants

Advances in transplantation and the growth of donor registries have provided greater options in the source of stem cells to be used in transplantation.

There are two categories of transplants: 

In an allogeneic transplant, the patient receives stem cells from the bone marrow or circulating blood cells from an immunologically compatible donor (as determined by Human Leukocyte Antigen testing, also known as HLA testing). A matched sibling donor is the ideal donor for an allogeneic transplant. Fortunately, most children who do not have an HLA matched donor are able to locate an unrelated donor through the National Donor Marrow Program.

Cord blood from an umbilical cord blood registry (or from the birth of a sibling) is the third possible source of stem cells for an allogeneic transplant.

In an autologous transplant, we harvest the patient’s own stem cells and then use them to reconstitute the immune system after very intensive chemotherapy and/or radiation.  We may use your child’s bone marrow as the source of the stem cells, or they can be collected by a relatively simple procedure called leukopheresis.  (If blood from your child’s umbilical cord was cryopreserved after birth, the cord blood can be used for autologous transplant.)