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
Leukemias and lymphomas, including:- 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
Data from the Center for International Blood and Marrow Transplant Research (CIBMTR) show the distribution of autologous and allogeneic transplants performed for diagnoses treated in 2005. (The data reflect only transplants reported to the CIBMTR, which represent approximately 35% to 39% of the estimated total number of transplants performed worldwide; 60% of those done in North America.)- Autologous and allogeneic transplants in North America (Figure 1)
- Allogeneic transplants worldwide (Figure 2)
Indications for Hematopoietic Stem Cell Transplants 2005 — North America. (CIBMTR data)
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Indications for Allogeneic Hematopoietic Stem Cell Transplants 2005 - Worldwide. (CIBMTR data)
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References
- 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.
