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
Hematologic disease
- Phagocyte disorders
  - Myelodysplasia
- Anemias
  - Paroxysmal nocturnal hemoglobinuria (PNH; severe aplasia)
  - Aplastic anemia
  - Acquired pure red cell aplasia
- Myeloproliferative disorders
Metabolic disorders
- Amyloidoses
  - Amyloid light chain (AL) amyloidosis
Environmentally-induced diseases
- Radiation poisoning
Viral diseases
- HTLV
- HIV^[1]

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)

References

↑ Hütter G, Nowak D, Mossner M, Ganepola S, Müßig A, Allers K, Schneider T, Hofmann J, Kücherer C, Blau O, Blau IW, Hofmann WK, Thiel E (2009). "Long-Term Control of HIV by CCR5 Delta32/Delta32 Stem-Cell Transplantation". N Engl J Med. 360 (7): 692–698. doi:10.1056/NEJMoa0802905. PMID 19213682.

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