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Review
. 2017 Nov 14;1(24):2281-2294.
doi: 10.1182/bloodadvances.2017009829.

The role of targeted therapy in the management of patients with AML

Affiliations
Review

The role of targeted therapy in the management of patients with AML

Alexander E Perl. Blood Adv. .

Abstract

Drug therapy for acute myeloid leukemia (AML) is finally undergoing major changes in 2017. This is due to the US Food and Drug Administration's approval of several new, targeted agents (midostaurin, enasidenib, and gemtuzumab ozogamicin). Paired with the recent approval of a novel liposomal formulation of daunorubicin/cytarabine (CPX-351/Vyxeos), the standard of care is changing rapidly in AML for subgroups. This review will focus on currently approved agents and promising novel agents in development and will highlight controversial areas in targeted treatment.

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Conflict of interest statement

Conflict-of-interest disclosure: A.E.P. is on the Board of Directors or an advisory committee for Asana Biosciences and Actinium Pharmaceuticals and has consulted for Daiichi Sankyo, Astellas, Novartis, Pfizer, Arog, Seattle Genetics, Asana Biosciences, and Actinium Pharmaceuticals. Off-label drug use: This presentation includes novel agents in clinical development that do not yet have label indications in acute myeloid leukemia (AML). The use of sorafenib, azacitidine, or decitabine for AML therapy is off label.

Figures

Figure 1.
Figure 1.
The flow diagram shows how integration of molecular diagnostics and NGS potentially can rapidly segregate patients into groups for whom particular and increasingly targeted therapies have shown benefit or are currently being tested. ATRA, all-trans retinoic acid; ATO, arsenic trioxide; CBF, core-binding factor; FISH, fluorescent in situ hybridization; GO, gemtuzumab ozogamicin; HMA, hypomethylating agent; MRC, myelodysplasia-related change; RT-PCR, reverse transcriptase-polymerase chain reaction; t-AML, therapy-related AML; 713, cytarabine by continuous infusion (7 doses) plus anthracycline (3 doses).

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