Table 2:

Selected key trials for approved agents for previously untreated acute myeloid leukemia

Author, year	Study arms	Study type, no. of patients	Patient characteristics	CR/CRi	OS*	Conclusion/Comments
Lancet 2018 [36]	CPX-351 vs 7+3	Phase III, 309	Age 60-75 yrs, s-AML or t-AML, MRC	38% vs 33% (p-0.1)	9.5 vs 5.9 month s (p-0.003)	OS better with CPX-351 in older, high-risk AML
DiNardo 2019 [42]	Venetoclax+Aza/Decitabine	Phase Ib, 145	Age ≥65 yrs, Unfit (25% s-AML)	67%	17.5 months	Venetoclax with aza or decitabine effective in older, unfit adults
Wei 2019 [43]	Venetoclax + LDAC	Phase I/II	Age ≥65 yrs, Unfit (49% s-AML)	54% s-AML-35% De novo AML-71%	10.1 months	Venetoclax with LDAC effective in older, unfit adults
Cortes 2019 [44]	LDAC with or without glasdegib	Phase II, 132	Age ≥ 55, Unfit, AML or high risk MDS	17% vs 2% (p<0.05)	8.3 vs 4.9 (p-0.003)	Glasdegib better in unfit or adults ≥75 years
Dombret 2015 [58]	Aza vs CCR (BSC, LDAC, or IC)	Phase III, 488	Age ≥65, Unfit intermediate-unfavorable risk cytogenetics, (10% s-AML)	28% vs 25%, p-0.5)	10.4 vs 6.5 months (p-0.1)	Aza better OS than BSC, similar to LDAC or IC†
Amadori 2016 [39]	GO vs BSC	Phase III, 237	Age ≥60, Unfit (31% s-AML)	27% with GO	4.9 vs 3.6 month s (p-0.005)	GO improves OS in favorable and intermediate risk cytogenetics

^*denotes median OS unless specified

^†Median OS in subgroup analysis: Aza vs BSC- 5.8 vs 3.7 p-0.02 ; Aza vs LDAC- 11.2 vs 6.4 p-0.4; Aza vs IC 13.3 vs 12.2 p-0.5

Aza- azacitidine, AML- acute myeloid leukemia, BSC- best supportive care, GO- gemtuzumab ozogamicin, IC- intensive chemotherapy, LDAC- low dose cytarabine, OS- overall survival, s-AML- secondary acute myeloid leukemia, t-AML- therapy related acute myeloid leukemia. 7+3-intensive induction regimen of cytarabine for 7 days with anthracycline for 3 days.