Table 1 Relation between NGS-defined... | American Society of Hematology

Table 1

Relation between NGS-defined cancer hotspot mutations and response to MLN4924/belinostat in primary AML samples (n = 16)

Patient	Gene	CDS_mut	AA_mut	Frequency, %	Viability, %*
Patient	Gene	CDS_mut	AA_mut	Frequency, %	Blast	LIC
1	ATM	c.998C>T	p.S333F	46	13.7	8.8
	FLT3	FLT3-ITD	FLT3-ITD	27
	NPM1	c.863_864insTCTG	p.W288fs*12	48
	SMARCB1	c.1119-41G>A	(Unknown)	51
2	FLT3	FLT3-ITD	FLT3-ITD	8	15.1	14.9
	HRAS	c.81T>C	p.H27H	50
	NPM1	c.863_864insTCTG	p.W288fs*12	43
3	KDR	c.1416A>T	p.Q472H	100	18.0	70.8
	KIT	c.1621A>C	p.M541L	49
	NRAS	c.34G>A	p.G12S	64
	PDGFRA	c.2472C>T	p.V824V	47
4	HRAS	c.81T>C	p.H27H	49	32.5	—
	KRAS	c.35G>A	p.G12D	8.2
	NRAS	c.181C>A	p.Q61K	8.5
5	HRAS	c.81T>C	p.H27H	48	39.1	59.7
	KIT	c.2447A>T	p.D816V	48
6	MET	c.1124A>G	p.N375S	6	43.9	40.1
	KRAS	c.35G>T	p.G12V	2
	FLT3	c.2504A>T	p.D835V	3
	STK11	c.1062C>G	p.F354L	42
7	TP53	c.722C>T	p.S241F	97	52.9	53.8
8	NRAS	c.35G>A	p.G12D	4	55.1	43.6
	PDGFRA	c.2472C>T	p.V824V	49
	KRAS	c.182A>C	p.Q61P	13
	KRAS	c.38G>A	p.G13D	15
9	BRAF	c.1799T>A	p.V600E	29	59.3	—
	SMARCB1	c.1119-41G>A	(Unknown)	50
10	MET	c.3029C>T	p.T1010I	50	60.8	9.2
	SMARCB1	c.1119-41G>A	(Unknown)	52
11	FLT3	c.2503G>G	p.D835H	57	69.6	—
	HRAS	c.81T>C	p.H27H	51
	IDH1	c.395G>A	p.R132H	48
	IDH1	c.315C>T	p.G105G	51
	KDR	c.1416A>T	p.Q472H	51
	NPM1	c.863_864insTCTG	p.W288fs*12	44
12	APC	c.4326T>A	p.P1442P	43	78.8	—
13	SMARCB1	c.1119-41G>A	(Unknown)	51	85.1	21.4
14	HRAS	c.81T>C	p.H27H	50	88.1	—
	IDH2	c.419G>A	p.R140Q	47
	STK11	c.1062C>G	p.F354L	49
15	IDH2	c.419G>A	p.R140Q	49	92.3	11.1
	NRAS	c.38G>A	p.G13D	42
	PDGFRA	c.2472C>T	p.V824V	47
	STK11	c.816C>T	p.Y272Y	100
	TP53	c.216_217delCCinsGT	p.P72R	52
16	NPM1	c.863_864TCTG	p.W288fs*12	49	94.4	—

Patient	Gene	CDS_mut	AA_mut	Frequency, %	Viability, %*
Patient	Gene	CDS_mut	AA_mut	Frequency, %	Blast	LIC
1	ATM	c.998C>T	p.S333F	46	13.7	8.8
	FLT3	FLT3-ITD	FLT3-ITD	27
	NPM1	c.863_864insTCTG	p.W288fs*12	48
	SMARCB1	c.1119-41G>A	(Unknown)	51
2	FLT3	FLT3-ITD	FLT3-ITD	8	15.1	14.9
	HRAS	c.81T>C	p.H27H	50
	NPM1	c.863_864insTCTG	p.W288fs*12	43
3	KDR	c.1416A>T	p.Q472H	100	18.0	70.8
	KIT	c.1621A>C	p.M541L	49
	NRAS	c.34G>A	p.G12S	64
	PDGFRA	c.2472C>T	p.V824V	47
4	HRAS	c.81T>C	p.H27H	49	32.5	—
	KRAS	c.35G>A	p.G12D	8.2
	NRAS	c.181C>A	p.Q61K	8.5
5	HRAS	c.81T>C	p.H27H	48	39.1	59.7
	KIT	c.2447A>T	p.D816V	48
6	MET	c.1124A>G	p.N375S	6	43.9	40.1
	KRAS	c.35G>T	p.G12V	2
	FLT3	c.2504A>T	p.D835V	3
	STK11	c.1062C>G	p.F354L	42
7	TP53	c.722C>T	p.S241F	97	52.9	53.8
8	NRAS	c.35G>A	p.G12D	4	55.1	43.6
	PDGFRA	c.2472C>T	p.V824V	49
	KRAS	c.182A>C	p.Q61P	13
	KRAS	c.38G>A	p.G13D	15
9	BRAF	c.1799T>A	p.V600E	29	59.3	—
	SMARCB1	c.1119-41G>A	(Unknown)	50
10	MET	c.3029C>T	p.T1010I	50	60.8	9.2
	SMARCB1	c.1119-41G>A	(Unknown)	52
11	FLT3	c.2503G>G	p.D835H	57	69.6	—
	HRAS	c.81T>C	p.H27H	51
	IDH1	c.395G>A	p.R132H	48
	IDH1	c.315C>T	p.G105G	51
	KDR	c.1416A>T	p.Q472H	51
	NPM1	c.863_864insTCTG	p.W288fs*12	44
12	APC	c.4326T>A	p.P1442P	43	78.8	—
13	SMARCB1	c.1119-41G>A	(Unknown)	51	85.1	21.4
14	HRAS	c.81T>C	p.H27H	50	88.1	—
	IDH2	c.419G>A	p.R140Q	47
	STK11	c.1062C>G	p.F354L	49
15	IDH2	c.419G>A	p.R140Q	49	92.3	11.1
	NRAS	c.38G>A	p.G13D	42
	PDGFRA	c.2472C>T	p.V824V	47
	STK11	c.816C>T	p.Y272Y	100
	TP53	c.216_217delCCinsGT	p.P72R	52
16	NPM1	c.863_864TCTG	p.W288fs*12	49	94.4	—

— represents CD34⁺/CD38⁻/CD123⁺ cells undetectable.

AA_mut, amino acid mutation; CDS_mut, coding DNA sequence mutation; LIC, CD34⁺/CD38⁻/CD123⁺ cells enriched for leukemia-initiating cells.

Cell viability after combined treatment with MLN4924 and belinostat, compared with untreated control, for which value is arbitrarily set as 100%.