Diseases Treated with BMT

A pediatric blood and marrow transplant (BMT) can be used to treat many types of malignant diseases (cancerous) and nonmalignant disorders (non-cancerous). 

Malignant Diseases: Blood Cancers

Blood cancers cause the bone marrow to make unhealthy numbers of cells. These cells crowd out the healthy cells and cause them to not work well. A BMT can help replace unhealthy blood stem cells in the bone marrow with healthy blood stem cells. The healthy blood stem cells help support the body and keep it healthy. They can also help keep the leukemia from coming back by using the immune system. 

Types of blood cancers eligible for BMT include:


  • Acute leukemia
    • Acute lymphocytic leukemia 
    • Acute myeloid leukemia 
    • Bi-phenotypic leukemia
  • Chronic leukemia
    • Chronic myeloid leukemia 
    • Juvenile myelomonocytic leukemia
  • Lymphoproliferative disorders
    • Non-Hodgkin's lymphoma 
    • Hodgkin's disease 

Malignant Diseases: Solid Tumor Cancers

If your child has a tumor that is hard to treat, doctors sometimes use high doses of chemotherapy as part of treatment. The high-dose chemotherapy can damage healthy blood stem cells. Your child may need a BMT to help rescue his blood stem cells after the chemotherapy.

Examples of solid tumors that may require a BMT include:

  • Neuroblastoma 
  • Certain brain tumors 
These tumors can be very aggressive cancers that resist regular chemotherapy. Without high-dose chemotherapy or radiation, these diseases may not be cured. High-dose treatment may increase the chance of a cure.

Non-Malignant Disorders (other than cancer)

Diseases other than childhood cancer may cause blood stem cells to not work properly. These problems may affect the white blood cells, red blood cells or platelets. In order to correct the blood cell problems, the entire bone marrow may need to be replaced with new blood stem cells.

Examples of these diseases are blood and genetic disorders, such as:


  • Stem cell disorders
    • Severe aplastic anemia 
    • Fanconi anemia
    • Paroxysmal nocturnal hemoglobinuria
    • Myelodysplastic syndromes
  • Phagocyte disorders
    • Chronic granulomatous disease
    • Leukocyte adhension deficiency
  • Histiocyte disorders
    • Familial erythrophagocytic lymphohistiocytosis
    • Hemophagocytic lymphohistiocytosis
  • Inherited erythrocyte abnormalities
  • Immune deficiency syndromes
    • Wiskott-Aldrich syndrome
    • Severe combined immune deficiency
  • Metabolic storage diseases
    • Hurler's syndrome
    • Adrenoleukodystrophy

    We are recognized as a national leader in transplanting children with sickle cell disease. Learn more about blood and marrow transplants (BMT), the only cure for sickle cell disease.

    In some cases, such as Tendoh’s, a BMT can cure sickle cell disease by replacing the sickle cells with healthy cells from a donor. Meet Tendoh.