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. Author manuscript; available in PMC: 2014 Oct 3.
Published in final edited form as: Semin Hematol. 2013 Oct 3;50(4):271–283. doi: 10.1053/j.seminhematol.2013.09.006

Table 4.

Novel Strategies for the Treatment of Adult AML

Agent Target/Class Comment
Fludarabine Nucleoside analog When used first line in the FLAG-Ida regimen, has been shown to be more effective than standard induction chemotherapy
Cladribine Nucleoside analog When added to 3+7 during induction, improved survival compared to 3+7 alone
Clofarabine Nucleoside analog Improves outcome when added to IA in patients younger than 40 yr
Gemtuzumab Monoclonal antibody Improves survival in subsets of younger and older patients when added to chemotherapy
Decitabine Hypomethylating agent Decitabine is approved in Europe in elderly patients based on improved survival compared to standard treatment; Extending the regimen to 10 days is a promising strategy; used prior to standard chemotherapy as epigenetic “priming” is an innovative approach
CPX-351 Liposomal formulation of cytarabine and daunorubicin High response rates noted in phase II trials, particularly in patients with secondary AML; also being studied in the salvage setting
Omacetaxine Protein synthesis inhibitor Improved outcomes in patients with favorable or intermediate cytogenetics compared to 7+3
FLT3 Inhibitors Tyrosine kinase Inhibitors Several promising oral agents being studied alone or in combination with chemotherapy or hypomethylating agents (midostaurin, sorafenib, quizartinib, crenolanib)
Vosaroxin DNA intercalating agent, topoisomerase II inhibitor Large, phase III study ongoing comparing moderate dose cytarabine with or without vosaroxin for relapsed AML