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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 2010:

  • Transplants in the United States, for patients ≤ 20 years (Figure 1)
  • Transplants in the United States (Figure 2)

Figure 1.
Indications for Hematopoietic Stem Cell Transplants in the United States, for patients ≤ 20 years, 2010. (CIBMTR data)

Indications for Hematopoietic Stem Cell Transplants in the United States, for patients ≤ 20 years, 2010
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Figure 2.
Indications for Hematopoietic Stem Cell Transplants in the United States, 2010. (CIBMTR data)

Indications for Hematopietic Stem Cell Transplants in the United States, 2010
View larger version 

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.