These are diseases for which Hematopoietic Stem Cell Transplants (HSCT) are a standard treatment.

For some diseases they are the only therapy, and in other diseases they are only employed when front-line therapies have failed or the disease is very aggressive. Most of the diseases for which HSCT is a standard treatment are disorders of blood cell lineage (see figure below), ranging from the stem cells in the bone marrow down to specific cell types in the blood. 


Inherited Diseases

Leukemia is a cancer of the blood immune system, whose cells are called leukocytes or white cells)

Acute Leukemia

Ø  Acute Lymphoblastic Leukemia (ALL)

Ø  Acute Myelogenous Leukemia (AML)

Ø  Acute Biphenotypic Leukemia

Ø  Acute Undifferentiated Leukemia

Chronic Leukemia

Ø  Chronic Myelogenous Leukemia (CML)

Ø  Chronic Lymphocytic Leukemia (CLL)

Ø  Juvenile Chronic Myelogenous Leukemia (JCML)

Ø  Juvenile Myelomonocytic Leukemia (JMML)

Inherited Red Cell (Erythrocyte) Abnormalities

Ø  Beta Thalassemia Major (also known as Cooley's Anemia)

Ø  Blackfan-Diamond Anemia

Ø  Pure Red Cell Aplasia

Ø  Sickle Cell Disease

Other Disorders of Blood Cell Proliferation


Ø  severe Aplastic Anemia

Ø  Congenital Dyserythropoietic Anemia

Ø  Fanconi Anemia  (Note: the first cord blood transplant in 1988 was for this disease)

Ø  Paroxysmal Nocturnal Hemoglobinuria (PNH)

Ø  Pure Red Cell Aplasia


Myelodysplastic Syndromes 
 Myelodysplasia is sometimes called pre-leukemia)

Ø  Refractory Anemia (RA)

Ø  Refractory Anemia with Ringed Sideroblasts (RARS)

Ø  Refractory Anemia with Excess Blasts (RAEB)

Ø  Refractory Anemia with Excess Blasts in Transformation (RAEB-T)

Ø  Chronic Myelomonocytic Leukemia (CMML)


Inherited Platelet Abnormalities (Platelets are small blood cells needed for clotting)

Ø  Amegakaryocytosis / Congenital Thrombocytopenia

Ø  Glanzmann Thrombasthenia

 Lymphoma is a cancer of the leukocytes that circulate in the blood and lymph vessels)

Ø  Hodgkin's Lymphoma

Ø  Non-Hodgkin's Lymphoma Burkitt's Lymphoma

Inherited Immune System Disorders -

Severe Combined Immunodeficiency (SCID)

Ø  SCID with Adenosine Deaminase Deficiency (ADA-SCID)

Ø  SCID which is X-linked

Ø  SCID with absence of T & B Cells

Ø  SCID with absence of T Cells, Normal B Cells

Ø  Omenn Syndrome


Inherited Immune System Disorders -Neutropenias

Ø  Kostmann Syndrome

Ø  Myelokathexis

Inherited Immune System Disorders - Other

Ø  Ataxia-Telangiectasia

Ø  Bare Lymphocyte Syndrome

Ø  Common Variable Immunodeficiency

Ø  DiGeorge Syndrome

Ø  Leukocyte Adhesion Deficiency

Ø  Lymphoproliferative Disorders (LPD)

Ø  Lymphoproliferative Disorder, X-linked (also known as Epstein-Barr Virus Susceptibility)

Ø  Wiskott-Aldrich Syndrome


Cancers in the bone marrow (Plasma Cell Disorders)

Ø  Multiple Myeloma

Ø  Plasma Cell Leukemia

Ø  Waldenstrom's Macroglobulinemia


Ø  Chediak-Higashi Syndrome

Ø  Chronic Granulomatous Disease

Ø  Neutrophil Actin Deficiency

Ø  Reticular Dysgenesis


Other cancers (Not originating in the blood system)

Ø  Neuroblastoma

Ø  Retinoblastoma

Myeloproliferative Disorders

Ø  Acute Myelofibrosis

Ø  Agnogenic Myeloid Metaplasia (Myelofibrosis)

Ø  Polycythemia Vera

Essential Thrombocythemia