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Acquired aplastic anemia is a clinical syndrome in which there is a deficiency of red cells, neutrophils, monocytes, and platelets in the blood, and fatty replacement of the marrow with a near absence of hematopoietic precursor cells. Reticulocytopenia is a constant feature. Neutropenia, monocytopenia, and thrombocytopenia, when severe, are life-threatening because of the risk of infection and bleeding, complicated by severe anemia. Most cases occur without an evident precipitating cause and are the result of autoreactive cytotoxic T lymphocytes that suppress or destroy primitive CD34+ multipotential hematopoietic cells. The disorder also can occur after (1) prolonged high-dose exposure to certain toxic chemicals (eg, benzene); (2) after specific viral infections (eg, Epstein-Barr virus [EBV]); (3) as an idiosyncratic response to certain pharmaceuticals (eg, ticlopidine, chloramphenicol); (4) as a feature of an autoimmune disorder (eg, lupus erythematosus); or, rarely, (5) in association with pregnancy. The final common pathway is through cytotoxic T-cell autoreactivity, whether idiopathic or associated with an inciting agent, because they all respond in a similar fashion to immunosuppressive therapy. The differential diagnosis of acquired aplastic anemia includes a hypoplastic marrow that can accompany paroxysmal nocturnal hemoglobinuria (PNH) or hypoplastic oligoblastic (myelodysplastic syndrome [MDS]) or polyblastic (acute) myelogenous leukemia. Allogeneic hematopoietic cell transplantation is curative in approximately 80% of younger patients with high-resolution human leukocyte antigen (HLA)–matched sibling donors, although the posttransplant period may be complicated by severe graft-versus-host disease. The disease may be significantly ameliorated or sometimes cured by immunotherapy, especially a regimen coupling antithymocyte globulin (ATG) with cyclosporine. However, after successful treatment with immunosuppressive agents, the disease may relapse or evolve into a clonal myeloid disorder, such as PNH, a clonal cytopenia, or oligoblastic or polyblastic myelogenous leukemia. The addition of eltrombopag (EPAG) to immunotherapy has increased the response rate and the quality of the response. Several uncommon inherited disorders, including Fanconi anemia (FA), Shwachman-Diamond syndrome, dyskeratosis congenita, and others have aplastic hematopoiesis as a primary manifestation.

Acronyms and Abbreviations:

A, adenine; ALG, antilymphocyte globulin; ALL, acute lymphocytic leukemia; AML, acute myelogenous leukemia; ATG, antithymocyte globulin; ATR, ataxia-telangiectasia mutated and rad3-related kinase; BFU-E, burst-forming unit–erythroid; CAMT, congenital (hereditary) amegakaryocytic thrombocytopenia; CD, cluster of differentiation; CFU-GM, colony-forming unit–granulocyte-macrophage; cMpl, thrombopoietin receptor; CMV, cytomegalovirus; DDT, dichlorodiphenyltrichloroethane; EBV, Epstein-Barr virus; EPAG, eltrombopag; ELF6, eukaryotic initiation factor 6; EPO, erythropoietin; G, guanine; G-CSF, granulocyte colony-stimulating factor; HIV, human immunodeficiency virus; HLA, human leukocyte antigen; IL, interleukin; JAK, Janus kinase; MDS, myelodysplastic syndrome; MRI, magnetic resonance imaging; NIH, National Institutes of Health; PCP, pentachlorophenol; PNH, paroxysmal nocturnal hemoglobinuria; ppm, parts per million; SCF, stem cell factor; TERC, telomerase RNA component; TERT, telomerase reverse transcriptase; Th1, T-cell helper type 1; TNF, tumor necrosis factor; TNT, trinitrotoluene; TPO, thrombopoietin.




Aplastic anemia is a clinical syndrome that results from a marked diminution of marrow blood cell production. The ...

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