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POTENTIALLY TREATABLE DISEASES WITH STEM CELL

Acute Leukemias

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

Inherited Erythrocyte Abnormalities

  • Beta Thalassemia Major
  • Blackfan-Diamond Anemia
  • Pure Red Cell Aplasia
  • Sickle Cell Disease

Chronic Leukemias

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

Phagocyte Disorders

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

Myelodysplastic Syndromes

  • Amyloidosis
  • Chronic Myelomonocytic Leukemia (CMML)
  • Refractory Anemia (RA)
  • Refractory Anemia with Excess Blasts (RAEB)
  • Refractory Anemia with Excess Blasts in Transformation (RAEB-T)
  • Refractory Anemia with Ringed Sideroblasts (RARS)

Histiocytic Disorders

  • Familial Erythrophagocytic
  • Lymphohistiocytosis
  • Hemophagocytosis
  • Histiocytosis-X
  • Langerhans' Cell Histiocytosis

Myeloproliferative Disorders

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

Congenital (Inherited) Immune System Disorders

  • Absence of T & B Cells SCID
  • Absence of T Cells, Normal B Cell SCID
  • Ataxia-Telangiectasia
  • Bare Lymphocyte Syndrome
  • Common Variable Immunodeficiency
  • DiGeorge Syndrome
  • Kostmann Syndrome
  • Leukocyte Adhesion Deficiency
  • Omenn's Syndrome
  • Severe Combined Immunodeficiency (SCID)
  • SCID with Adenosine Deaminase
  • Deficiency
  • Wiskott-Aldrich Syndrome
  • X-Linked Lymphoproliferative Disorder

Lymphoproliferative Disorders

  • Hodgkin's Disease
  • Non-Hodgkin's Lymphoma
  • Prolymphocytic Leukemia

Inherited Metabolic Disorders

  • Adrenoleukodystrophy
  • Alpha Mannosidosis
  • Beta-Glucuronidase Deficiency (MPS-Vll)
  • Gaucher’s Disease
  • Hunter’s Syndrome (MPS-ll)
  • Hurler’s Syndrome (MPS-lH)
  • Krabbe Disease
  • Maroteaux-Lamy Syndrome (MPS-Vl)
  • Metachromatic Leukodystrophy
  • Morquio Syndrome (MPS-lV)
  • Mucolipidosis II (l-cell Disease)
  • Mucopolysaccharidoses (MPS)
  • Niemann-Pick Disease
  • Sanfilippo Syndrome (MPS-lll)
  • Scheie Syndrome (MPS-lS)
  • Sly Syndrome
  • Wolman Disease

Stem Cell Disorders

  • Aplastic Anemia (Severe)
  • Congenital Cytopenia
  • Dyskeratosis Congenita
  • Fanconi Anemia
  • Paroxysmal Nocturnal Hemoglobinuria (PNH)

Plasma Cell Disorders

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

Inherited Platelet Abnormalities

  • Amegakaryocytosis / Congenital Thrombocytopenia

Other Inherited Disorders

  • Cartilage-Hair Hypoplasia
  • Ceroid Lipofuscinosis
  • Congenital Erythropoietic Porphyria
  • Glanzmann Thrombasthenia
  • Lesch-Nyhan Syndrome
  • Osteopetrosis
  • Tay Sachs Disease