'Acute myeloid leukemia: a comprehensive review and 2016 update'

'Acute myeloid leukemia: a comprehensive review and 2016 update'

1 July 2016 | I De Kouchkovsky and M Abdul-Hay
Acute Myeloid Leukemia (AML) is the most common acute leukemia in adults, with an incidence of over 20,000 cases per year in the United States alone. AML is characterized by the accumulation of poorly differentiated myeloid cells due to chromosomal translocations and mutations in genes involved in hematopoietic proliferation and differentiation. The disease is highly heterogeneous, with cases stratified into favorable, intermediate, and adverse-risk groups based on cytogenetic profiles. Recurrent genetic mutations, such as FLT3-ITD, NPM1, and CEBPA, have helped refine prognosis and guide management. Despite advancements in supportive care, the backbone of therapy remains a combination of cytarabine- and anthracycline-based regimens, with allogeneic stem cell transplantation for eligible candidates. Elderly patients often cannot tolerate such regimens and have a particularly poor prognosis. Recent advances in treatment include novel agents like FLT3-ITD inhibitors, STAT3 inhibitors, IDH1/IDH2 inhibitors, and clofarabine, as well as monoclonal antibodies and chimeric antigen receptor (CAR) T-cell therapy. These treatments offer promise for improved outcomes, particularly in elderly patients and those with relapsed or refractory diseases. However, the molecular diversity of AML means that a single "magic bullet" is unlikely, and the development of new treatments in concert with improved genetic profiling and risk stratification is expected to result in incremental gains in remission and survival.Acute Myeloid Leukemia (AML) is the most common acute leukemia in adults, with an incidence of over 20,000 cases per year in the United States alone. AML is characterized by the accumulation of poorly differentiated myeloid cells due to chromosomal translocations and mutations in genes involved in hematopoietic proliferation and differentiation. The disease is highly heterogeneous, with cases stratified into favorable, intermediate, and adverse-risk groups based on cytogenetic profiles. Recurrent genetic mutations, such as FLT3-ITD, NPM1, and CEBPA, have helped refine prognosis and guide management. Despite advancements in supportive care, the backbone of therapy remains a combination of cytarabine- and anthracycline-based regimens, with allogeneic stem cell transplantation for eligible candidates. Elderly patients often cannot tolerate such regimens and have a particularly poor prognosis. Recent advances in treatment include novel agents like FLT3-ITD inhibitors, STAT3 inhibitors, IDH1/IDH2 inhibitors, and clofarabine, as well as monoclonal antibodies and chimeric antigen receptor (CAR) T-cell therapy. These treatments offer promise for improved outcomes, particularly in elderly patients and those with relapsed or refractory diseases. However, the molecular diversity of AML means that a single "magic bullet" is unlikely, and the development of new treatments in concert with improved genetic profiling and risk stratification is expected to result in incremental gains in remission and survival.
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