cord blood banking

diseases currently using standard treatment with stem cells

Cord blood glossary

These are diseases treated with stem cells where transplants of blood-forming stem cells (hematopoietic stem cell transplants, HSCT) are a standard treatment. For some diseases they are the only therapy, and in other diseases they are only employed when front-line therapies have failed, or the disease is very aggressive. Most of the diseases for which HSCT is a standard treatment are disorders of blood cell lineage, ranging from the stem cells in the bone marrow down to specific cell types in the blood.

Diseases for Which Standard Treatments Are Currently Being Used:

Leukemias

– Acute leukemia

  • Acute lymphoblastic leukemia – ALL
  • Acute myelogenous leukemia – AML
  • Acute biphenotypic leukemia
  • Acute undifferentiated leukemia

– Chronic leukemia

  • Chronic myelogenous leukemia – CML
  • Chronic lymphocytic leukemia – CLL
  • Juvenile chronic myelogenous leukemia – JCML
  • Juvenile myelomonocytic leukemia – JMML

– Myelodysplastic syndromes

  • Refractory anemia – RA
  • Refractory anemia with ringed sideroblasts – RARS
  • Refractory anemia with excess blasts – RAEB
  • Refractory anemia with excess blasts in transformation – RAEB-T
  • Chronic myelomonocytic leukemia – CMML

– Lymphomas

  • Non-Hodgkin’s lymphoma
  • Hodgkin’s lymphoma

Other disorders of blood cell proliferation

– Anemias

  • Aplastic anemia
  • Congenital dyserythropoietic anemia
  • Fanconi anemia*
  • Paroxysmal nocturnal hemoglobinuria – PNH
  • Pure red cell aplasia

– Inherited red cell (erythrocyte) abnormalities

  • Beta thalassemia major – Cooley’s anemia
  • Blackfan-Diamond anemia
  • Pure red cell aplasia
  • Sickle cell disease

– Inherited platelet abnormalities

  • Amegakaryocytosis / Congenital thrombocytopenia
  • Glanzmann thrombasthenia

– Myeloproliferative disorders

  • Acute myelofibrosis
  • Agnogenic myeloid metaplasia – myelofibrosis
  • Polycythemia vera
  • Essential thrombocythemia

– Inherited immune system disorders

  • Severe combined immunodeficiency – SCID
  • SCID with absence of T cells, normal B cells
  • SCID with adenosine deaminase deficiency – ADA-SCID
  • SCID with absence of T & B cells
  • Omenn syndrome
  • SCID which is X-linked

– Inherited immune system disorders – neutropenias

  • Kostmann syndrome
  • Myelokathexis

– Other inherited immune system disorders

  • Ataxia-Telangiectasia
  • Leukocyte adhesion deficiency
  • DiGeorge syndrome
  • Bare lymphocyte syndrome
  • Common variable immunodeficiency
  • Wiskott-Aldrich syndrome
  • Lymphoproliferative disorders LPD
  • Lymphoproliferative disorder, X-linked – aka Epstein-Barr virus susceptibility
  • Hemophagocytic lymphohistiocytosis
  • Wiskott-Aldrich syndrome

– Bone marrow cancers

  • Multiple myeloma
  • Plasma cell leukemia
  • Waldenstrom’s macroglobulinemia

– Other cancers – Not originating in the blood system

  • Neuroblastoma
  • Retinoblastoma

– Other inherited immune system disorders

  • Cartilage-hair hypoplasia
  • Gunther’s disease erythropoietic porphyria
  • Hermansky-Pudlak syndrome
  • Pearson’s syndrome
  • Shwachman-Diamond syndrome
  • Systemic mastocytosis

 – Transplants for Inherited Metabolic Disorders

  • Mucopolysaccharidoses – MPS
  • Hurler’s syndrome – MPS-IH
  • Scheie syndrome – MPS-IS
  • Hunter’s syndrome – MPS-II
  • Sanfilippo syndrome – MPS-III
  • Morquio syndrome – MPS-IV
  • Maroteaux-Lamy syndrome MPS-VI
  • Sly syndrome, beta-glucuronidase deficiency MPS-VII
  • Mucolipidosis II – I-cell disease

– Leukodystrophy disorders

  • Adrenoleukodystrophy – ALD
  • Adrenomyeloneuropathy – AMN
  • Krabbe disease – globoid cell leukodystrophy
  • Metachromatic leukodystrophy
  • Pelizaeus-Merzbacher disease

– Lysosomal storage diseases

  • Niemann-Pick disease
  • Sandhoff disease
  • Wolman disease

– Other inherited metabolic disorders

  • Lesch-Nyhan syndrome (case report)
  • Osteopetrosis