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- W2193659829 abstract "Aplastic anemia, an uncommon hematological disease, is the paradigm of the human bone marrow failure syndromes. The pathophysiology is immune mediated in most cases, with activated type 1 cytotoxic T cells implicated. The molecular basis of the aberrant immune response and deficiencies in hematopoietic cells is now being defined genetically; examples are telomere repair gene mutations in the target cells and dysregulated T-cell activation pathways. Almost universally fatal just a few decades ago, aplastic anemia can now be cured or ameliorated by stem-cell transplantation or immunosuppressive drug therapy. Immunosuppression with antithymocyte globulins (ATGs) and cyclosporine is effective at restoring blood-cell production in the majority of patients, but relapse and especially evolution of clonal hematologic diseases remain problematic. Allogeneic stem-cell transplant from histocompatible sibling donors is curative in the great majority of young patients with severe aplastic anemia; the major challenges are extending the benefits of transplantation to patients who are older or who lack family donors.KeywordsAplastic anemiapancytopeniaCD34+" @default.
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- W2193659829 date "2008-01-01" @default.
- W2193659829 modified "2023-10-16" @default.
- W2193659829 title "Idiopathic Aplastic Anemia" @default.
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- W2193659829 doi "https://doi.org/10.1007/978-1-60327-285-8_95" @default.
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