Diseases Treatable by Stem Cell Transplant

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Acute Leukemias
Acute Lymphoblastic Leukemia (ALL)
Acute Myelogenous Leukemia (AML)

Chronic Leukemias
Chronic Myelogenous Leukemia (CML)
Juvenile Myelomonocytic Leukemia (JMML)

Myelodysplastic Syndromes
Refractory Anemia (RA) (all types)
Myelofibrosis
Chronic Myelomonocytic Leukemia (CMML)

Stem Cell Disorders
Aplastic Anemia (Severe)
Fanconi Anemia
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Pure Red Cell Aplasia
Congenital Cytopenias
Dyskeratosis Congenita

Histiocytic Disorders
Hemophagocytoic Lymphohistiocytosis (HLH)
Langerhans Cell Histiocytosis (LCH)

Inherited Platelet Abnormalities
Amegakaryocytosis/Congenital Thrombocytopenia

Multiple Myeloma and other Plasma Cell Disorders
Plasma Cell Leukemia
Waldenstrom’s Macroglobulinemia

Potential Future Applications
Alzheimer’s Disease
Diabetes
Heart Disease
Liver Disease
Muscular Dystrophy
Parkinson’s Disease
Spinal Cord Injury
Stroke
Chronic Granulomatus Disease Disorder
Neutrophil Actin Deficiency
Reticular Dysgenesis

Lymphoproliferative Disorders
Non-Hodgkin Lymphoma
Prolymphocytic Leukemia
Hodgkin's Disease

Phagocyte Disorders, including
Chediak-Higashi Syndrome
Chronic Granulomatous Disease

Inherited Erythrocyte Abnormalities
Beta Thalassemia Major
Sickle Cell Disease
Blackfan-Diamond Anemia
Pure Red Cell Aplasia

Inherited Metabolic Disorders/Liposomal Storage Diseases, including
Hurler’s Syndrome (MPS-IH)
Adrenoleukodystrophy
Metachromatic Leukodystrophy

Other Inherited Disorders, including
Glanzmann Thrombasthenia
Osteopetrosis

Autoimmune Disorders, including
Evans’ Syndrome
Multiple Sclerosis (experimental)
Rheumatoid Arthritis (experimental)
Systemic Lupus Erythematosus (experimental)

Inherited Immune System Disorders, including
Kostmann Syndrome
DiGeorge Syndrome
Severe Combined Immunodeficiency (SCID)
Wiskott-Aldrich Syndrome
X-Linked Lymphoproliferative Disorder

Other Malignancies
Brain Tumors
Ewing’s Sarcoma
Neuroblastoma
Ovarian Cancer
Testicular Cancer