Table 1 Selected emerging therapies for the management of AML

 Liposomal formulation of 7 + 3

CPX-351

≥60 years, sAML, fit for induction therapy

Single agent

3

[10–15]

 Topoisomerase II inhibitor

Vosaroxin

≥60 years, R/R

With cytarabine

3

[19]

 DNMT inhibitor

Guadecitabine

Unfit for intensive therapy or R/R

Single agent

3

[24, 25]

 IDH1 inhibitor

AG-120, IDH305, FT-2102

IDH1 mutated

Single agent/with AZA or induction and consolidation

1b/2

[31, 33]

 IDH2 inhibitor

Enasidenib (AG-221)

IDH2 mutated

Single agent/with AZA or induction and consolidation

3

[34]

 HDAC inhibitors

Panobinostat

Ongoing investigation

With HMAs or induction and consolidation

1b/2

[38, 39]

Vorinostat

3

[40–43]

Entinostat

2

[45]

Pracinostat

2

[46]

 BET inhibitor

OTX015

Ongoing investigation

Single agent

1

[47]

 DOT1L inhibitor

Pinometostat

MLL-rearranged

Single agent

1

[49]

 LSD1 inhibitor

Tranylcypromine, GSK2879552, ORY-1001

Ongoing investigation

Single agent/with ATRA

1/2

–

FLT3 inhibitors

Sorafenib

FLT3-ITD-mutated R/R

With AZA

2

[60]

Midostaurin

FLT3-ITD or FLT3-TKD, ≤60 years

With induction and consolidation

3

[63]

Quizartinib

FLT3-ITD-mutated R/R

Single agent

3

[66–68]

Crenolanib

FLT3-ITD or FLT3-TKD

Single agent/with induction and consolidation

2

[71–73]

Gilteritinib

FLT3-ITD or FLT3-TKD R/R

Single agent

3

[74]

 Anti-CD33

Vadastuximab (SGN-CD33A)

CD33+

Single agent/with HMAs or induction and consolidation

3

[75–77]

 Polo-like kinase inhibitor

Volasertib

Unfit for intensive therapy

With LDAC or decitabine

3

[78]

 BCL-2 inhibitor

Venetoclax (ABT-199)

R/R, or older (≥65 years) and unfit for intensive therapy

Single agent/with HMAs or LDAC

2

[82, 83]

 Aminopeptidase inhibitor

Tosedostat

≥60 year, R/R or unfit for intensive therapy

Single agent/with cytarabine or HMAs

2

[84–87]