Recent developments in acute myelogenous leukemia therapy.

Recent progress has been made in several areas in the treatment of acute myelogenous leukemia (AML): prognostic factors, allogeneic bone marrow transplantation, and new and targeted therapies. Delineation and clarification of prognostic factors have led to improved risk determination, with research moving from cytogenetics to an examination of molecular markers. Trends in the area of allogeneic bone marrow transplantation include broad adoption of reduced-intensity conditioning despite the lack of prospective comparative studies. Although the preponderance of data has established this as a feasible option, a true understanding of how much of an advantage it conveys needs to be established in prospective studies. The use of alternative donors is another advance, and recent data are promising, but survival is poor if transplantation is performed when disease is active, especially during refractory relapse or refractory disease. When haploidentical matched donors are used, survival rates appear similar to those reported with matched unrelated-donor transplants. Analysis of the data for allogeneic transplantation shows that HLA-identical sibling transplants to patients in the first complete remission (CR1) provide the highest probability of long-term survival, compared with HLA-identical sibling transplants to patients in later remissions. Similarly, unrelated-donor transplants to high-risk patients in CR1 lead to a greater degree of success than unrelated-donor transplants to patients in CR2 or later remission. Cord blood has also been established as a suitable source for hematopoietic transplantation in AML. A third area of recent progress involves new and targeted therapies. Multiple new agents with tremendous potential are in development and clinical trials. Therapy can even be tailored to several specific genetic subtypes of AML.