Diseases Treated

Hematopoietic Stem Cell Transplantation (HSCT) is a standard treatment for the diseases listed below. 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, ranging from the stem cells in the bone marrow down to specific cell types in the blood.  Not all of the diseases below have been treated with hematopoietic stem cells from cord blood. Other sources of hematopoietic stem cells include the placenta, bone marrow and peripheral blood.


Acute Leukemia

  • Acute Lymphoblastic Leukemia
  • Acute Myelogenous Leukemia
  • Acute Biphenotypic Leukemia
  • Acute Undifferentiated Leukemia

Chronic Leukemia

  • Chronic Myelogenous Leukemia
  • Chronic Lymphocytic Leukemia
  • Juvenile Chronic Myelogenous Leukemia
  • Juvenile Myelomonocytic Leukemia

Myelodysplastic Syndromes (sometimes called pre-leukemia)

  • Refractory Anemia
  • Refractory Anemia with Ringed Sideroblasts
  • Refractory Anemia with Excess Blasts
  • Refractory Anemia with Excess Blasts in Transformation
  • Chronic Myelomonocytic Leukemia


  • Hodgkin’s Lymphoma
  • Non-Hodgkin’s Lymphoma Burkitt’s Lymphoma

Inherited Red Cell (Erythrocyte) Abnormalities

  • Beta Thalassemia Major (also known as Cooley’s Anemia)
  • Blackfan-Diamond Anemia
  • Pure Red Cell Aplasia
  • Sickle Cell Disease

Other Disorders of Blood Cell Proliferation


  • Severe Aplastic Anemia
  • Congenital Dyserythropoietic Anemia
  • Fanconi Anemia
  • Paroxysmal Nocturnal Hemoglobinuria
  • Pure Red Cell Aplasia

Inherited Platelet Abnormalities

  • Amegakaryocytosis / Congenital Thrombocytopenia
  • Glanzmann Thrombasthenia

Myeloproliferative Disorders

  • Acute Myelofibrosis
  • Agnogenic Myeloid Metaplasia (Myelofibrosis)
  • Polycythemia Vera
  • Essential Thrombocythemia

Inherited Immune System Disorders

  • Severe Combined Immunodeficiency (SCID)
  • SCID with Adenosine Deaminase Deficiency
  • SCID which is X-linked
  • SCID with absence of T & B Cells
  • SCID with absence of T Cells, Normal B Cells
  • Omenn Syndrome

Inherited Immune System Disorders – Neutropenias

  • Kostmann Syndrome
  • Myelokathexis

Inherited Immune System Disorders – Other

  • Ataxia-Telangiectasia
  • Bare Lymphocyte Syndrome
  • Common Variable Immunodeficiency
  • DiGeorge Syndrome
  • Leukocyte Adhesion Deficiency
  • Lymphoproliferative Disorders
  • Lymphoproliferative Disorder, X-linked (also known as Epstein-Barr Virus Susceptibility)
  • Wiskott-Aldrich Syndrome

Phagocyte Disorders

  • Chediak-Higashi Syndrome
  • Chronic Granulomatous Disease
  • Neutrophil Actin Deficiency
  • Reticular Dysgenesis

Cancers in the Bone Marrow

  • Multiple Myeloma
  • Plasma Cell Leukemia
  • Waldenstrom’s Macroglobulinemia

Solid Tumors

  • Neuroblastoma
  • Retinoblastoma

Transplants for Inherited Disorders Affecting the Immune System & Other Organs

  • Cartilage-Hair Hypoplasia
  • Gunther’s Disease (Erythropoietic Porphyria)
  • Hermansky-Pudlak Syndrome
  • Pearson’s Syndrome
  • Shwachman-Diamond Syndrome
  • Systemic Mastocytosis

Transplants for Inherited Metabolic Disorders

  • Mucopolysaccharidoses Storage Diseases
  • Mucopolysaccharidoses (MPS)
  • Hurler’s Syndrome (MPS-IH)
  • Scheie Syndrome (MPS-IS)
  • Hunter’s Syndrome (MPS-II)
  • Sanfilippo Syndrome (MPS-III)
  • Morquio Syndrome (MPS-IV)
  • Maroteaux-Lamy Syndrome (MPS-VI)
  • Sly Syndrome, Beta-Glucuronidase Deficiency (MPS-VII)
  • Mucolipidosis II (I-cell Disease)

Leukodystrophy Disorders

  • Adrenoleukodystrophy (ALD)/Adrenomyeloneuropathy (AMN)
  • Krabbe Disease (Globoid Cell Leukodystrophy)
  • Metachromatic Leukodystrophy
  • Pelizaeus-Merzbacher Disease

Lysosomal Storage Diseases

  • Gaucher Disease
  • Niemann-Pick Disease
  • Sandhoff Disease
  • Tay-Sachs Disease
  • Wolman Disease

Inherited Disorders – Other

  • Lesch-Nyhan Syndrome
  • Osteopetrosis

Source: Parent’s Guide to Cord Blood Foundation as of March 07, 2010. Please visit their web site for the most current and comprehensive information.