Dramatic disappearance of the marrow and profound cytopenia seemingly occurring without prodromal signs have stimulated the research of generations of physicians and scientists since the initial descriptions of aplastic anemia (AA) in the last decades of the nineteenth century. The almost inevitable fatal outcome of severe AA made it a subject of research by renowned hematologists. Later, its illustrative features allowed insights into stem cell function, regulation of blood cell production, and the role of immune system in the regulation of hematopoiesis. Research into the causes of AA has led to the description of cellular elements of blood, and later to the introduction of stem cell transplantation as a means of replacing a defective stem cell compartment. Today, the longer survival of patients with AA uncovers new instructive aspects of the disease and provides new therapeutic challenges. These aspects include the evolution of clonal diseases, such a myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH).

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