Stem cell transplant is being used to treat many types of conditions and can often be used when conventional treatments have been unsuccessful. Stem cell transplantation is an emerging first-line therapy for some serious diseases, including:

  • Hematologic malignancies and leukemias particularly:
    • Acute lymphoblastic leukemia (ALL)
    • Acute myelogenous leukemia (AML)
    • Chronic myelogenous leukemia (CML)
    • Juvenile myelomonocytic leukemias (JMML/CMML)
    • Myelodysplastic syndromes (MDS)
    • Secondary leukemias
    • Relapsed/refractory leukemia that has failed other therapies
  • Hodgkin and Non-Hodgkin lymphoma
  • Neuroblastoma
  • Sarcomas
  • Other solid tumors of childhood
  • Hemophagocytic lymphohistiocytosis (HLH)
  • Hemoglobinopathies including sickle cell anemia and β-thalassemia
  • Aplastic anemia
  • Fanconi anemia
  • Rare blood diseases including chronic granulomatous disease, Chediak-Higashi and Schwachman-Diamond syndromes, and dyskeratosis congenital disease
  • Paroxysmal nocturnal hemoglobinuria
  • Bone marrow failure syndromes
  • Metabolic/lysosomal storage diseases
  • Immunodeficiencies including severe combined immunodeficiency (SCID) and Wiskott-Aldrich Syndrome
  • Autoimmune conditions including systemic lupus erythematosus (SLE), rheumatoid arthritis and scleroderma