Table 2:

Newly-Approved AML Therapies

Therapy	Indication	Key Outcomes	Key Adverse Events	Ref#
Midostaurin	Newly diagnosed FLT3+ AML	Per the CALGB 10603/RATIFY trial, median OS of 74.7 months in the midostaurin group versus 25.6 months in placebo group	Grade 3 or greater rash/desquamation and nausea	[20]
CPX-351	Newly diagnosed therapy-related AML or AML with myelodysplastic-related changes	Phase III trial comparing CPX-351 with 7+3 chemotherapy showed superior OS (9.56 months versus 5.95 months) in the CPX-351 treatment arm, as well as CR and CRi response rates (47.7% versus 33.3%)	Hemorrhage, neutropenia, hypersensitivity reactions	[30]
Gemtuzumab ozogamicin	Newly diagnosed CD33+ AML or R/R CD33+ AML	The 2012 ALFA-0701 study compared low fractionated-dose GO with standard first-line chemotherapy, and found an improvement in median OS in the GO arm (34 months versus 19.2 months, p = 0.046)	Transaminitis, and veno-occlusive disease (rare but severe complication)	[24]
Enasidenib	IDH2-mutant R/R AML	CR or CRi in 26.6% of patients, with an additional 12% of patients with partial response for an overall response rate of 38.5%. Median OS was 9.3 months overall, with a median OS of 19.7 months in patients with CR	Hyperleukocytosis, and IDH-inhibitor-associated differentiation syndrome (IDH-DS)	[31]

Abbreviations: FLT3, fms like tyrosine kinase 3; AML, acute myeloid leukemia; OS, overall survival; CR, complete remission; CRi, complete remission with incomplete hematologic response; R/R, relapsed/refractory; IDH2, isocitrate dehydrogenase 2.