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BACKGROUND: General therapeutic options for patients with acute myelogenous leukemia (AML) are reviewed and specific new therapies are described. METHODS: Data in this review came from the published literature and the M. D. Anderson Cancer Center's acute leukemia database. RESULTS: Outcome following standard therapy of AML is so variable that is best to speak of a range of outcomes determined by various prognostic factors. Therapy can (and usually does) fail because of treatment-induced mortality or (more usually) resistance to therapy. Performance status and age are the principal predictors of early death, whereas cytogenetics, a history of abnormal blood counts, and MDR1 expression are predictors of resistance. Using this information, physicians can categorize patients into those in whom 1) standard therapy is indicated, 2) either standard or investigational therapy is appropriate, and 3) investigational therapy is indicated. The majority of even newly diagnosed patients belong to Group 3. The availability of allogeneic or autologous transplantation does not alter this conclusion. Investigational therapies have been developed that are directed against the CD33 surface antigen, the multidrug-resistant MDR1 protein, and other targets. Because of the number of new therapies clinical research in AML should emphasize pilot trials rather than traditionally large Phase III studies. CONCLUSIONS: Most patients with newly diagnosed AML should be offered investigational regimens.
Elihu H. Estey (Mon,) studied this question.