Bone marrow failure is a general term covering many diseases. Bone marrow, the sponge-like tissue found inside bones, contains blood-forming stem cells that develop into red blood cells, white blood cells, and platelets. Disorders affecting the stem cells can, in turn, lead to bone marrow failure-rare, potentially life-threatening diseases in which the bone marrow stops functioning or produces abnormal blood cells. These diseases are classified into two major categories: acquired bone marrow failure and inherited bone marrow failure. Acquired bone marrow failure may be caused by a variety of factors including exposure to certain chemicals, environmental toxins, viruses, or by autoimmune responses. Acquired bone marrow failure diseases include aplastic anemia, myelodysplasia, paroxysmal nocturnal hemoglobinuria and pure red cell aplasia. Inherited forms of bone marrow failure arise from specific alterations or abnormalities of genes. The most common inherited bone marrow failure disorders include Fanconi anemia, dyskeratosis congenita, Shwachman-Diamond syndrome, and Diamond-Blackfan anemia. Two treatment options for bone marrow failure disorders include drug therapy (i.e., corticosteroids, androgens, and hematopoietic growth factors) and hematopoietic stem cell transplant. Treatment of bone marrow failure is determined by the cause and severity of the illness; for some patients, the currently available treatment options may not be appropriate or feasible.
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