title: Clonal evolution of aplastic anaemia to myelodysplasia/acute myeloid leukaemia and paroxysmal nocturnal haemoglobinuria.
abstract: Aplastic anaemia (AA) is a non-malignant haemopoietic disorder characterised by peripheral blood pancytopenia and a hypocellular bone marrow. Successful management of acquired AA including treatment with immunosuppressive agents, mainly antithymocyte globulin (ATG) and cyclosporin or allogeneic haemopoietic stem cell transplantation, has resulted in long-term survival of many patients. The later evolution of complicating clonal disorders such as paroxysmal nocturnal haemoglobinuria, myelodysplasia and acute myeloid leukaemia in patients treated with immunosuppressive therapy may be a manifestation of the natural history of the aplasia, the development of which may or may not be increased by immunosuppressive therapy. A persistent, profound deficiency and/or defect in the stem cell compartment, despite haematological recovery after immunosuppressive therapy, may create an unstable situation which predisposes to later clonal disorders. A review of the progression of AA to clonal disorders is now outlined.
authors.affiliation: Department of Haematology, St George's Hospital Medical School, London, UK.
authors.forename: J A
authors.initials: JA
authors.lastname: Tooze
authors.forename: J C
authors.initials: JC
authors.lastname: Marsh
authors.forename: E C
authors.initials: EC
authors.lastname: Gordon-Smith
mesh_terms: D000208:Acute Disease; D000741:Anemia, Aplastic / Q000097:blood / Q000503:physiopathology* / Q000628:therapy; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D001853:Bone Marrow / Q000473:pathology; D016179:Granulocyte Colony-Stimulating Factor / Q000009:adverse effects; D006457:Hemoglobinuria, Paroxysmal / Q000097:blood / Q000503:physiopathology*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D007951:Leukemia, Myeloid / Q000097:blood / Q000503:physiopathology*; D009190:Myelodysplastic Syndromes / Q000097:blood / Q000503:physiopathology*; D010198:Pancytopenia
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D016179:Granulocyte Colony-Stimulating Factor