Tcr Vbeta Repertoire Analyses In Childhood Aquired Servere Aplastic Anemia: A New Predictor For Ist Response?

Acquired Severe Aplastic Anemia (aSAA) is a rare disease characterized by pancytopenia and bone marrow hypocellularity. There is good clinical and laboratory evidence that a T-cell mediated autoimmune mechanism directed against stem- and progenitor cells located in bone marrow plays a major role in the pathogenesis of aSAA. Immunosuppressive therapy (IST) and bone marrow transplantation (BMT) are the two treatment options. One of the key issues selecting the appropriate treatment is the long latency period of 4–6 months before a response to IST. In a large multicenter prospective study in children we could show that the severity of the disease (i.e. degree of granulocytopenia