Standard chemotherapy can cure only a fraction (30-40%) of younger and very few older patients with acute
myeloid leukemia (AML). While conventional allografting can extend the cure rates, its application remains limited
mostly to younger patients and those in remission. Limited efficacy of current therapies and improved understanding of
the disease biology provided a spur for clinical trials examining novel agents and therapeutic strategies in AML. Clinical
studies with novel chemotherapeutics, antibodies, different signal transduction inhibitors, and epigenetic modulators demonstrated
their clinical activity; however, it remains unclear how to successfully integrate novel agents either alone or in
combination with chemotherapy into the overall therapeutic schema for AML. Further studies are needed to examine their
role in relation to standard chemotherapy and their applicability to select patient populations based on recognition of
unique disease and patient characteristics, including the development of predictive biomarkers of response. With increasing
use of nonmyeloablative or reduced intensity conditioning and alternative graft sources such as haploidentical donors
and cord blood transplants, the benefits of allografting may extend to a broader patient population, including older AML
patients and those lacking a HLA-matched donor. We will review here recent clinical studies that examined novel pharmacologic
and immunologic approaches to AML therapy.
Keywords: Acute myeloid leukemia, chemotherapy, novel agents, transplant.
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