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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 lymphoma
  • Non-Hodgkin 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)

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

Indications for Blood and Marrow Transplantation in North America, 2005
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Figure 2.
Indications for Allogeneic Hematopoietic Stem Cell Transplants 2005 - Worldwide. (CIBMTR data)

Indications for Allogeneic Blood and Marrow Transplantation, 2005 - Worldwide
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The National Marrow Donor Program® (which operates Be The Match®) and its research arm, the CIBMTR, collect and study data to learn how to improve patient outcomes.

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.