Current Stem Cell Applications | ||
· Bone Marrow Failure Disorders
o Amegakaryocytosis
o Aplastic Anemia (Severe)
o Blackfan-Diamond Anemia
o Congenital Cytopenia*
o Congenital Dyserythropoietic Anemia
o Dyskeratosis Congenita
o Fanconi Anemia
o Paroxysmal Nocturnal Hemoglobinuria (PNH)
o Pure Red Cell Aplasia
· Hemoblobinopathies
o Beta Thalassemia Major
o Sickle Cell Disease
· Histiocytic Disorders
o Familial Erythrophagocytic Lymphohistiocytosis
o Hemophagocytosis
o Langerhans’ Cell Histiocytosis (Histiocytosis X)
· Inherited Immune System Disorders
o Chronic Granulomatous Disease
o Congenital Neutropenia
o Leukocyte Adhesion Deficiency
o Severe Combined Immunodeficiencies (SCID) including:
§ Adenosine Deaminase Deficiency*
§ Bare Lymphocyte Syndrome
§ Chediak-Higashi Syndrome*
§ Kostmann Syndrome
§ Omenn Syndrome
§ Purine Nucleoside Phosphorylase Deficiency
§ Reticular Dysgenesis
o Wiskott-Aldrich Syndrome
o X-Linked Lymphoproliferative Disorder
· Inherited Metabolic Disorders
o Adrenoleukodystrophy
o Fucosidosis
o Gaucher Disease*
o Hunter Syndrome (MPS-II)
o Hurler Syndrome (MPS-IH)
o Krabbe Disease
o Lesch-Nyhan Syndrome
o Mannosidosis*
o Maroteaux-Lamy Syndrome (MPS-VI)
o Metachromatic Leukodystrophy
o Mucolipidosis II (I-cell Disease)*
o Neuronal Ceroid Lipofuscinosis (Batten Disease)*
o Niemann-Pick Disease*
o Sandhoff Disease*
o Sanfilippo Syndrome (MPS-III)
o Scheie Syndrome (MPS-IS)
o Sly Syndrome
o Tay Sachs*
o Wolman Disease
· Leukemias and Lymphomas
o Acute Biphenotypic Leukemia*
o Acute Lymphocytic Leukemia (ALL)
o Acute Myelogenous Leukemia (AML)
o Acute Undifferentiated Leukemia*
o Adult T Cell Leukemia/Lymphoma
o Chronic Lymphocytic Leukemia (CLL)
o Chronic Myelogenous Leukemia (CML)
o Hodgkin’s Lymphoma
o Juvenile Chronic Myelogenous Leukemia (JCML)
o Juvenile Myelomonocytic Leukemia (JMML)
o Myeloid/Natural Killer (NK) Cell Precursor Acute Leukemia
o Non-Hodgkin’s Lymphoma
o Polymphocytic Leukemia
· Myelodysplastic/Myeloproliferative Disorders
o Acute Myelofibrosis*
o Agnogenic Myeloid Metaplasia (Myelofibrosis)*
o Amyloidosis
o Chronic Myelomonocytic Leukemia (CMML)
o Essential Thrombocythemia*
o Polycythemia Vera*
o Refractory Anemias (RA) including:
§ Refractory Anemia with Excess Blasts (RAEB)
§ Refractory Anemia with Excess Blasts in Transformation (RAEB-T)
§ Refractory Anemia with Ringed Sideroblasts (RARS)
· Plasma Cell Disorders
o Multiple Myeloma
o Plasma Cell Leukemia
o Waldenstrom’s Macroglobulinemia
o Other Inherited Disorders
o Cartilage-Hair Hypoplasia
o Congenital Erythropoietic Porphyria (Gunther Disease)
o DiGeorge Syndrome
o Osteopetrosis
· Other Malignancies
o Brain Tumors**
o Ewing Sarcoma*
o Neuroblastoma
o Ovarian Cancer**
o Renal Cell Carcinoma**
o Rhabdomyosarcoma
o Small Cell Lung Cancer**
o Testicular Cancer**
o Thymoma (Thymic Carcinoma)
· Other
o Chronic Active Epstein Barr
o Evans Syndrome
o Multiple Sclerosis**
o Rheumatoid Arthritis**
o Systemic Lupus Erythematosus**
o Thymic Dysplasia
*Refer to http://www.clinicaltrials.gov/for additional information
**Not routinely eligible for participation in CBR’s Designated Transplant
Program Source: Medical literature and http://www.clinicaltrials.gov/
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Emerging Stem Cell Applications
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· Diabetes
· Heart Disease
· Liver Disease
· Muscular Dystrophy
· Parkinson’s Disease
· Spinal Cord Injury
· Stroke
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