List of conditions treated with hematopoietic stem cell transplantation

Hematopoietic stem cell transplantation may be used to treat a number of conditions both congenital and acquired.

Acquired

• Malignancies
  • Hematological
    • Leukemias
      • Acute lymphoblastic leukemia (ALL)
      • Acute myeloid leukemia (AML)
      • Chronic lymphocytic leukemia (CLL)
      • Chronic myelogenous leukemia (CML), accelerated phase or blast crisis
    • Lymphomas
      • Hodgkin's disease
      • Non-Hodgkin's lymphoma
    • Myelomas
      • Multiple myeloma (Kahler's disease)
  • Solid tumor cancers
    • Neuroblastoma
    • Desmoplastic small round cell tumor
    • Ewing's sarcoma
    • Choriocarcinoma
• Hematologic disease
  • Phagocyte disorders
    • Myelodysplasia
  • Anemias
    • Paroxysmal nocturnal hemoglobinuria (PNH; severe aplasia)
    • Aplastic anemia
    • Acquired pure red cell aplasia
  • Myeloproliferative disorders
    • Polycythemia vera
    • Essential thrombocytosis
    • Myelofibrosis
• Metabolic disorders
  • Amyloidoses
    • Amyloid light chain (AL) amyloidosis
• Environmentally-induced diseases
  • Radiation poisoning
• Viral diseases
  • HTLV
  • HIV^[1]
• Autoimmune diseases
  • Multiple sclerosis^[2]

Congenital

• Lysosomal storage disorders
  • Lipidoses (disorders of lipid storage)
    • Neuronal ceroid lipofuscinoses
      • Infantile neuronal ceroid lipofuscinosis (INCL, Santavuori disease,)
      • Jansky–Bielschowsky disease (late infantile neuronal ceroid lipofuscinosis)
    • Sphingolipidoses
      • Niemann–Pick disease
      • Gaucher disease
    • Leukodystrophies
      • Adrenoleukodystrophy
      • Metachromatic leukodystrophy
      • Krabbe disease (globoid cell leukodystrophy)
  • Mucopolysaccharidoses
    • • Hurler syndrome (MPS I H, α-L-iduronidase deficiency)
      • Scheie syndrome (MPS I S)
      • Hurler–Scheie syndrome (MPS I H-S)
      • Hunter syndrome (MPS II, iduronidase sulfate deficiency)
      • Sanfilippo syndrome (MPS III)
      • Morquio syndrome (MPS IV)
      • Maroteaux–Lamy syndrome (MPS VI)
      • Sly syndrome (MPS VII)
  • Glycoproteinoses
    • • Mucolipidosis II (I-cell disease)
      • Fucosidosis
      • Aspartylglucosaminuria
      • Alpha-mannosidosis
  • Other
    • • Wolman disease (acid lipase deficiency)
• Immunodeficiencies
  • T-cell deficiencies
    • Ataxia-telangiectasia
    • DiGeorge syndrome
  • Combined T- and B-cell deficiencies
    • Severe combined immunodeficiency (SCID), all types
  • Well-defined syndromes
    • Wiskott–Aldrich syndrome
  • Phagocyte disorders
    • Kostmann syndrome
    • Shwachman–Diamond syndrome
  • Immune dysregulation diseases
    • Griscelli syndrome, type II
  • Innate immune deficiencies
    • NF-Kappa-B Essential Modulator (NEMO) deficiency (Inhibitor of Kappa Light Polypeptide Gene Enhancer in B Cells Gamma Kinase deficiency)
• Hematologic diseases
  • • Hemoglobinopathies
    • Sickle cell disease
    • β thalassemia major (Cooley's anemia)
  • Anemias
    • Aplastic anemia
      • Diamond–Blackfan anemia
      • Fanconi anemia
  • Cytopenias
    • Amegakaryocytic thrombocytopenia
  • Hemophagocytic syndromes
    • Hemophagocytic lymphohistiocytosis (HLH)