CURRENT CONCEPTS IN THE PATHOPHYSIOLOGY AND TREATMENT OF APLASTIC ANEMIA Running head: Current concepts in aplastic anemia
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Review in translational hematology Current concepts in the pathophysiology and treatment of aplastic anemia
Aplastic anemia, an unusual hematologic disease, is the paradigm of the human bone marrow failure syndromes. Almost universally fatal just a few decades ago, aplastic anemia can now be cured or ameliorated by stem-cell transplantation or immunosuppressive drug therapy. The pathophysiology is immune mediated in most cases, with activated type 1 cytotoxic T cells implicated. The molecular basis o...
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Historically viewed in isolation as an odd, rare, and invariably fatal blood disease, aplastic anemia is now of substantial interest for its immune pathophysiology, its relationship to constitutional BM failure syndromes and leukemia, and the success of both stem cell transplantation and immunosuppressive therapies in dramatically improving survival of patients. Once relegated to a few presenta...
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Acquired aplastic anemia (AA) is the typical bone marrow failure syndrome characterized by an empty bone marrow; an immune-mediated pathophysiology has been demonstrated by experimental works as well as by clinical observations. Immunusuppressive therapy (IST) is a key treatment strategy for aplastic anemia; since 20 years the standard IST for AA patients has been anti-thymocyte globuline (ATG)...
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Aplastic anemia is a rare disease affecting 1—2 people per one million of the population. The hallmark of the disease is peripheral pancytopenia with hypo cellular bone marrow in the absence of infiltrative disease to the bone marrow. It is classified into moderate, severe, and very severe aplastic anemia based on actual cell count. Damage to the stem cells in the bone marrow is central to the ...
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The treatment and medical management of aplastic anemia fundamentally differ between patients with inherited versus acquired marrow failure; however, the diagnosis of an inherited bone marrow failure syndrome is frequently obscure. Recent exciting advances in our understanding of the molecular pathophysiology of the inherited bone marrow failure syndromes have resulted in a profusion of new tes...
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