Diseases Treatable by Hematopoietic Cell Transplant

An estimated 45,000 to 50,000 hematopoietic cell transplants (bone marrow, PBSC, or cord blood transplants — BMT) are performed annually worldwide to treat patients with life-threatening malignant and non-malignant diseases. [1] Due to advances in transplantation, long-term experience and ongoing clinical trials, patient eligibility for transplant continues to expand and the list of indications for which transplant may be a standard treatment option continues to develop.

Below is a list of diseases for which autologous or allogeneic hematopoietic cell transplant may be a treatment. The list includes diagnoses for which transplant is a standard treatment as well as diagnoses for which the role of transplant is a somewhat newer option. The current distribution of transplants performed by diagnosis follows.

Diseases treatable

• Acute myelogenous leukemia
• Acute lymphoblastic leukemia
• Chronic myelogenous leukemia
• Chronic lymphocytic leukemia
• Juvenile myelomonocytic leukemia
• Hodgkin's lymphoma
• Non-Hodgkin's lymphoma

Multiple myeloma and other plasma cell disorders

Severe aplastic anemia and other marrow failure states, including:

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

SCID and other inherited immune system disorders, including:

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

Hemoglobinopathies, including:

• Beta thalassemia major
• Sickle cell disease

Hurler's syndrome and other inherited metabolic disorders, including:

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

Myelodysplastic and myeloproliferative disorders, including:

• Refractory anemia (all types)
• Chronic myelomonocytic leukemia
• Agnogenic myeloid metaplasia (myelofibrosis)

Familial erythrophagocytic lymphohistiocytosis and other histiocytic disorders

Other malignancies

Current transplant indications

• Autologous and allogeneic transplants in North America (Figure 1)
• Allogeneic transplants worldwide (Figure 2)

Figure 1.
Indications for Hematopoietic Stem Cell Transplants 2005 — North America. (CIBMTR data)


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Figure 2.
Indications for Allogeneic Hematopoietic Stem Cell Transplants 2005 - Worldwide. (CIBMTR data)


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References

1. Horowitz MM. Uses and Growth of Hematopoietic Cell Transplantation. In: Blume KG, Forman SJ, Appelbaum FR, eds. Thomas' Hematopoietic Cell Transplantation. 3rd ed. Malden, Mass: Blackwell; 2004:9-15.