Targeted ITP strategies: Do they elucidate the biology of ITP and related disorders?

An elusive goal in hematology is a detailed understanding of autoimmune blood disorders including idiopathic (immune) thrombocytopenic purpura (ITP), autoimmune hemolytic anemia (AlHA), and Evans Syndrome. While it is clear that these disorders have an autoimmune pathophysiology, the exact mechanisms are not clear. Agents that target the immune system specifically, such as rituximab and tacrolimus, are currently being used clinically with favorable results. Targeted therapy may be useful not only in treating these difficult diseases, but also may provide clues to the underlying disease pathophysiology. Carefully designed studies using targeted therapy may provide biologic predictors of disease severity, as well as predictors of response to therapy. Pediatr Blood Cancer 2006;47:706-709. (c) 2006 Wiley-Liss, Inc.