Harnessing the benefits of available targeted therapies in acute myeloid leukaemia Review


Authors: Kantarjian, H.; Short, N. J.; DiNardo, C.; Stein, E. M.; Daver, N.; Perl, A. E.; Wang, E. S.; Wei, A.; Tallman, M.
Review Title: Harnessing the benefits of available targeted therapies in acute myeloid leukaemia
Abstract: Research has resulted in regulatory approval of nine agents for acute myeloid leukaemia indications by the US Food and Drug Administration since 2017: the Bcl-2 inhibitor, venetoclax; two FLT3 inhibitors, midostaurin and gilteritinib; two IDH inhibitors, ivosidenib (IDH1 inhibitor) and enasidenib (IDH2 inhibitor); the anti-CD33 antibody–drug conjugate, gemtuzumab ozogamicin; the oral, poorly absorbable hypomethylating agent, azacitidine; the liposomal formulation of cytarabine and daunorubicin (5:1 ratio), CPX-351; and the hedgehog signalling pathway inhibitor, glasdegib. A 100% absorbable oral formulation of the hypomethylating agent decitabine was approved for the treatment of myelodysplastic syndrome and chronic myelomonocytic leukaemia, and might be used as an alternative to parenteral hypomethylating agents. Several of the approvals are as single-agent therapies or in specific combinations for narrow indications, thus offering poor treatment value. In this Review, we discuss ongoing research into combinations containing these commercially available targeted therapies for acute myeloid leukaemia. © 2021 Elsevier Ltd
Journal Title: The Lancet Haematology
Volume: 8
Issue: 12
ISSN: 2352-3026
Publisher: Elsevier Science, Inc.  
Date Published: 2021-12-01
Start Page: e922
End Page: e933
Language: English
DOI: 10.1016/s2352-3026(21)00270-2
PROVIDER: scopus
PUBMED: 34687602
PMCID: PMC8996707
DOI/URL:
Notes: Review -- Export Date: 1 December 2021 -- Source: Scopus
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  1. Eytan Moshe Stein
    342 Stein
  2. Martin Stuart Tallman
    649 Tallman