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Diseases Treated with Cord Blood

Today there are more than 80 diseases and conditions being treated with cord blood stem cells and the list continues to grow.

Blood Disorders

Acute Myelofibrosis 

Agnogenic Myeloid Metaplasia (Myelofibrosis)


Aplastic Anemia (Severe)

Beta Thalassemia Major

Blackfan-Diamond Anemia

Congenital Amegakaryocytic Thrombocytopenia (CAT)

Congenital Cytopenia

Congenital Dyserythropoietic Anemia

Dyskeratosis Congenita

Essential Thrombocythemia

Fanconi Anemia

Glanzmann’s Thrombasthenia

Myelodysplastic Syndrome

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Polycythemia Vera

Pure Red Cell Aplasia

Refractory Anemia with Excess Blasts (RAEB)

Refractory Anemia with Excess Blasts in Transition (RAEB-T)

Refractory Anemia with Ringed Sideroblasts (RARS)

Shwachman-Diamond Syndrome

Sickle Cell Disease



Acute Biphenotypic Leukemia

Acute Lymphocytic Leukemia (ALL)

Acute Myelogenous Leukemia (AML)

Acute Undifferentiated Leukemia

Adult T Cell Leukemia/Lymphoma

Chronic Active Epstein Barr

Chronic Lymphocytic Leukemia (CLL)

Chronic Myelogenous Leukemia (CML)

Chronic Myelomonocytic Leukemia (CMML)

Ewing Sarcoma

Hodgkin’s Lymphoma

Juvenile Chronic Myelogenous Leukemia (JCML)

Juvenile Myelomonocytic Leukemia (JMML)​

Myeloid/Natural Killer (NK) Cell PrecursorAcute Leukemia

Non-Hodgkin’s Lymphoma

Prolymphocytic Leukemia

Plasma Cell Leukemia

Leukocyte Adhesion Deficiency

Multiple Myeloma



Thymoma (Thymic Carcinoma)

Waldenstrom’s Macroglobulinemia

Wilms Tumor



Immune Disorders

Adenosine Deaminase Deficiency (SCID)

Bare Lymphocyte Syndrome (SCID)

Chediak-Higashi Syndrome (SCID)

Chronic Granulomatous Disease

Congenital Neutropenia

DiGeorge Syndrome

Evans Syndrome


Hemophagocytic Lymphohistiocytosis (HLH)

Hemophagocytosis Langerhans’ Cell Histiocytosis (Histiocytosis X)

IKK Gamma Deficiency (NEMO Deficiency)

Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-linked (IPEX) Syndrome

Kostmann Syndrome (SCID)


Omenn Syndrome (SCID)

Phosphorylase Deficiency (SCID)

Purine Nucleoside (SCID)

Reticular Dysgenesis (SCID)

Severe Combined Immunodeficiency Diseases (SCID)

Thymic Dysplasia

Wiskott-Aldrich Syndrome

X-linked Agammaglobulinemia

X-Linked Hyper IgM Syndrome

X-Linked Lymphoproliferative Disorder


Metabolic Disorders

Congenital Erythropoietic Porphyria (Gunther Disease)

Gaucher Disease

Hunter Syndrome (MPS-II)

Hurler Syndrome (MPS-IH)

Krabbe Disease

Lesch-Nyhan Syndrome


Maroteaux-Lamy Syndrome (MPS-VI)

Metachromatic Leukodystrophy

Mucolipidosis II (I-cell Disease)

Neuronal Ceroid Lipofuscinosis (Batten Disease)

Niemann-Pick Disease

Sandhoff Disease

Sanfilippo Syndrome (MPS-III)

Scheie Syndrome (MPS-IS)

Sly Syndrome (MPS-VII)

Tay Sachs

Wolman Disease

X-Linked Adrenoleukodystrophy


Note: Some diseases may be known by other names. The use of umbilical cord blood stem cells to treat many diseases and conditions is typically considered standard therapy, while the treatment for other conditions may still be in a clinical trial phase. Currently, stem cells are being used in trials for the treatment of brain injuries, cerebral palsy, autism, diabetes, spinal cord injuries, heart disease, liver disease, and others. These conditions have not been included in this list.

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