Diseases Treated

List of Standard Diseases Treatable with Stem Cells
Referenced by the National Marrow Donor Program

The list includes diagnoses for which transplant is a standard treatment as well as diagnoses for which the role of transplant is a newer option¹. Essentially stem cells from all three sources (bone marrow, mobilized peripheral blood and umbilical cord blood) can and have been used to treat these groups of diseases².

Leukaemias and lymphomas

Cancer of the lymphocytes, a type of blood cell that forms part of the immune system, also called “cancer of the blood”.

  • Acute lymphoblastic leukaemia
  • Acute myelogenous leukaemia
  • Chronic lymphocytic leukaemia
  • Chronic myelogenous leukaemia
  • Hodgkin lymphoma
  • Non-Hodgkin lymphoma
  • Juvenile myelomonocytic leukaemia
  • Hodgkin lymphoma
  • Non-Hodgkin lymphoma
  • Juvenile myelomonocytic leukaemia

 

Multiple myeloma and other plasma cell disorders
Cancer of the plasma cells in the bone marrow.

Severe aplastic anaemia and other marrow failure states

A group of disorders that result in the failure of normal functioning of cells in the bone marrow./td>

  • Amegakaryocytosis/congenital
  • thrombocytopenia
  • Fanconi anaemia
  • Paroxysmal nocturnal hemoglobinuria(PNH)
  • Pure red cell aplasia
  • Severe aplastic anaemia

SCID and other inherited immune system disorders

Diseases in which children are born without an immune system or with an incomplete immune system.

  • Severe combined immunodeficiency (SCID, all sub-types)
  • Wiskott-Aldrich syndrome

Hemoglobinopathies

Genetic defects that result in abnormal structure of one of the globin chains of the haemoglobin molecule that

is responsible for carrying of oxygen in the blood.

  • Beta thalassemia major
  • Sickle cell disease

Hurler’s Syndrome and other inherited metabolic disorders

A group of inherited diseases in which the lack of an enzyme affects various organs and tissues, including the

brain. Enzymes are proteins that play many roles, including metabolizing (breaking down) substances in the

body.

  • Adrenoleukodystrophy
  • Hurler’s syndrome (MPS-IH)
  • Metachromatic leukodystrophy

Myelodysplastic and myeloproliferative disorders

A group of diseases that affect the bone marrow and blood. It can also develop into a fast-growing, severe

leukaemia called acute myelogenous leukaemia

  • Agnogenic myeloid metaplasia(myelofibrosis)
  • Chronic myelomonocytic leukaemia
  • Refractory anaemia (all types)

Familial erythrophagocytic lymphohistiocytosis and other histiocytic disorders

Diseases that occur when there is an over-production of white blood cells known as histiocytes that can lead to

organ damage and tumour formation.

Other malignancies

References:

National Marrow Donor Program 

Broxmeyer, Hal E.  Enhancing engraftment of cord blood cells via insight intothe biology of stem/progenitor cell function. Annals of the New York Academy of Sciences. Issue: Hematopoietic Stem Cells VIII

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