Abstract
Bone marrow failure syndromes, characterized by a failure of the bone marrow hematopoietic function, may be congenital (Fanconi Anemia and Blackfand Diamond Anemia) or acquired, and they can affect selectively one of the series (erythroblastopenia, leukopenia or thrombocytopenia) or the whole hematopoiesis (aplasia). Congenital forms are often, although not always, associated with malformations and they have an increased risk of malignancies. The acquired forms are usually of immune origin and idiopathic, but associated diseases that require specific therapy must be discarded. The only curative treatment of bone marrow aplasia, both acquired or congenital, is hematopoietic transplantation, but its indication has to de assessed individually, because of the high morbidity and mortality of the procedure. In aplasias of congenital origin, specific gene therapy is under investigation.
Published Version
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