Table 1.

Summary of clinical activity of venetoclax combination regimens in treatment-naïve and relapsed/refractory AML

Combination	Ven mg	Phase/Study	Population	N	Age [range]	CR/CRi rate	MRD (-)	DOR	OS
LDAC [19]	600	I/II	Frontline	82		54% ND 71% s-AML 35%		8.1 m 11.6m 8.1 m	10.1 m
HMA [20]	400–1200	Ib	Frontline	145	74 [65–86]	67% ND 67% s-AML 67% VEN 400 73% VEN 800 65%	29%	11.3 m 12.5 m NR 12.5 m 11 m	17.5m 12.5 m NR NR 17.5 m
5+2 [30]	50–600	Ib	Frontline	44	72 [63–80]	71% ND 95% s-AML 42%
LDAC [66]	100–200	Retrospective	Frontline AML, MDS, CMML	19	77 [64–87]	52.6%			NR 61% at 6m
DAC-10 [29]	200–400	II	Frontline R/R AML	48	>60	R/R 44% ND 71% s-AML 71%	50% 52% 40%	3.3 m NR NR	NR NR NR
HMA [34]	50–800	Retrospective	Frontline R/R AML	42	67 [25–88]	43% ND 33% s-AML 50% R/R 40%			6.2m 5.8 m 5.8 m 6.6 m
HMA [28]	200–400	Retrospective	Frontline R/R AML	107	68 [19–86]	53% ND 61% R/R 48%	64%		12.5 m 14.6 m (CR)
HMA/LDAC [35]	400–800	Retrospective	R/R AML	24	66 [29–85]	28.6%	9.5%		72% at 3 m.
HMA/LDAC [33]	100–800	Retrospective	R/R AML, MDS, BPDCN	43	68 [25–83]	21%			3 m 4.8 m (CR)
HMA/LDAC [36]	100–800	Prospective	R/R AML	22	76 [41–92]	41%			5.5 m 12.5 m (CR)
FLAG-IDA [31]	200	Ib/II	R/R AML	12	49 [32–72]	73%		NR	67% at 6 m.
Cobimetinib or idasanutlin[55]	400–800	Ib	R/R AML s-AML	42	72 [60–92]	Cobimetinib 18% Idasanutlin 20%		1–5 m 1.3–6.7 m

Abbreviations: AML, acute myeloid leukemia; Ven, venetoclax; CR, complete response; CRi, complete response with incomplete hematological recovery; MRD, minimal residual disease; DOR, duration of response; OS, overall survival; LDAC, low-dose cytarabine; ND, newly diagnosed; s-AML, secondary AML; m, months; HMA, hypomethylating agent; NR, not reached; 5+2, 5-day cytarabine and 2-day idarubicin; MDS, myelodysplastic syndrome; CMML, chronic myelomonocytic leukemia; DAC-10, 10-day decitabine, R/R, relapsed/refractory; BPDCN, blastic plasmocytoid dendritic cell neoplasm; FLAG-IDA, fludarabine, cytarabine, idarubicin, granulocyte colony–stimulating factor.