TY - JOUR
T1 - Harnessing the benefits of available targeted therapies in acute myeloid leukaemia
AU - Kantarjian, Hagop
AU - Short, Nicholas J.
AU - DiNardo, Courtney
AU - Stein, Eytan M.
AU - Daver, Naval
AU - Perl, Alexander E.
AU - Wang, Eunice S.
AU - Wei, Andrew
AU - Tallman, Martin
N1 - Funding Information:
This work is supported in part by the Charif Souki Cancer Research Grant, the MD Anderson Cancer Center Leukemia specialized program of research excellence ] (CA100632), and the Cancer Center Support Grant ( P30CA016672 ). AHW acknowledges support from the Leukemia & Lymphoma Society Specialized Center of Research (Strasser) and the Medical Research Future Fund of Australia.
Publisher Copyright:
© 2021 Elsevier Ltd
PY - 2021/12
Y1 - 2021/12
N2 - 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.
AB - 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.
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U2 - 10.1016/S2352-3026(21)00270-2
DO - 10.1016/S2352-3026(21)00270-2
M3 - Review article
C2 - 34687602
AN - SCOPUS:85119606704
SN - 2352-3026
VL - 8
SP - e922-e933
JO - The Lancet Haematology
JF - The Lancet Haematology
IS - 12
ER -