Diseases Treated

Diseases Treated

Stem cell transplantation has become standard treatment for almost 80 diseases. For some diseases, stem cell transplantation is first-line therapy. For others, stem cells are employed only when first-line therapies have failed or the disease is very aggressive.

There are different types of stem cells. Those currently used for transplantation are called hematopoietic stem cells (HSCs). HSCs are able to transform into cells found in the blood: red cells, white cells, and platelets. They replace diseased cells with healthy, new cells and restore the body’s ability to make blood.

Because HSCs transform into blood cells, stem cell transplantation is mostly used to treat diseases of the blood and bone marrow (where blood cells are made). HSCs can treat

  • Cancers, such as leukemia. In leukemia treatment, stem cells replace stem cells in bone marrow that have been destroyed by chemotherapy or radiation
  • Severe blood diseases (such as sickle cell anemia) in which the body doesn’t make enough red blood cells, or they don’t work well
  • Immune-deficiency diseases in which the body loses the ability to make some types of white blood cells

 Below is a full list of diseases for which stem cell transplantation is a standard treatment.

Leukemias

Acute Leukemia

  • Acute Lymphoblastic Leukemia (ALL)
  • Acute Myelogenous Leukemia (AML)
  • Acute Biphenotypic Leukemia
  • Acute Undifferentiated Leukemia

Chronic Leukemia

  • Chronic Myelogenous Leukemia (CML)
  • Chronic Lymphocytic Leukemia (CLL)
  • Juvenile Chronic Myelogenous Leukemia (JCML)
  • Juvenile Myelomonocytic Leukemia (JMML)

Myelodysplastic Syndromes (sometimes called pre-leukemia)

  • Refractory Anemia
  • Refractory Anemia with Ringed Sideroblasts (Sideroblastic Anemia)
  • Refractory Anemia with Excess Blasts
  • Refractory Anemia with Excess Blasts in Transformation
  • Chronic Myelomonocytic Leukemia (CMML)

 Lymphomas

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

 Other Disorders of Blood Cell Proliferation

Anemias

  • Aplastic Anemia
  • Congenital Dyserythropoietic Anemia
  • Fanconi Anemia
  • Paroxysmal Nocturnal Hemoglobinuria (PNH)

Inherited Red Cell Abnormalities

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

Inherited Platelet Abnormalities

  • Amegakaryocytosis / Congenital Thrombocytopenia
  • Glanzmann Thrombasthenia

Inherited Immune System Disorders: Severe Combined Immunodeficiency (SCID)

  • SCID with Adenosine Deaminase Deficiency (ADA-SCID)
  • 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

  • Infantile Genetic Agranulocytosis (Kostmann Syndrome)
  • Myelokathexis

Inherited Immune System Disorders: Other

  • Ataxia-Telangiectasia
  • Bare Lymphocyte Syndrome
  • Common Variable Immunodeficiency
  • DiGeorge Syndrome
  • Hemophagocytic Lymphohistiocytosis
  • Leukocyte Adhesion Deficiency

Lymphoproliferative Disorders

  • Lymphoproliferative Disorder, X-linked (Epstein-Barr Virus Susceptibility)
  • Wiskott-Aldrich Syndrome

Myeloproliferative Disorders

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

Phagocyte Disorders

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

Bone Marrow Cancers

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

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

Leukodystrophy Disorders

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

Lysosomal Storage Diseases

  • Niemann-Pick Disease
  • Sandhoff Disease
  • Wolman Disease

Inherited Disorders: Other

  • Lesch-Nyhan Syndrome
  • Osteopetrosis

Solid Tumors Not Originating in the Blood or Immune System

  • Neuroblastoma
  • Medulloblastoma
  • Retinoblastoma

Source: Parent’s Guide to Cord Blood FoundationTM as of November 19, 2014. Please visit the Parent’s Guide website for the most current and comprehensive information on cord blood therapy.