TABLE 3.

Presenting characteristics, molecular analysis and outcome for participants with T-ALL on DFCI 05-001 and 11-001

	N (%)	5-yr DFS%* [95% CI]	p-value	5-yr EFS% [95% CI]	p-value	5-yr OS% [95% CI]	p-value
No. Pts.	123			81 [73-87]		90 [83-94]
Age
<10 yrs.	67 (54)			83 [72-90]	0.49	91 [80-96]	0.75
≥10 yrs.	56 (46)			78 [64-87]		89 [78-95]
WBC
< 50K	53 (43)			79 [64-88]	0.60	92 [81-97]	0.48
≥ 50K	70 (57)			83 [72-90]		88 [78-94]
Sex
Male	91 (74)			81 [72-88]	0.97	90 [81-95]	0.99
Female	32 (26)			80 [60-90]		91 [74-97]
CNS Status
CNS 1	71 (58)			87 [76-93]	0.052	94 [85-98]	0.17
CNS 2	42 (34)			76 [60-86]		86 [71-95]
CNS 3	10 (8)			60 [25-83]		80 [41-93]
Mediastinal Mass
Yes	55 (45)			85 [73-92]	0.27	89 [77-95]	0.77
No	68 (55)			77 [65-86]		91 [81-96]
ETP Phenotype
Yes	100 (81)			54 [29-74]	0.0006	85 [61-95]	0.31
No	21 (17)			87 [79-92]		92 [84-96]
Unknown	2 (2)			---		----
End-induction MRD
<10−3	69 (75)	94 [85-98]	0.079	---		94 [85-98]	0.24
≥10−3	23 (25)	79 [51-92]		---		86 [65-96]
<10−4	45 (49)	98 [85-99]	0.031	---		98 [85-99]	0.055
≥10−4	47 (51)	84 [69-92]		---		87 [73-94]
Post-Induction Asparaginase†
E.coli	59 (48)			93 [83-97]	0.68	93 [82-97]	0.81
Pegaspargase	37 (30)			88 [72-95]		92 [76-97]
Erwinia	2 (2)			100 [NA]		100 [NA]
Calaspargase pegol	9 (7)			100 [NA]		100 [NA]
Gene/Pathway Mutations‡
NOTCH1 gene
Present	59 (51)	92 [80-97]	0.84	86 [73-93]	0.26	95 [85-98]	0.080
Absent	56 (49)	91 [79-97]		78 [65-87]		85 [72-92]
Notch Pathway§
Present	66 (57)	90 [78-95]	0.39	84 [72-91]	0.43	92 [83-97]	0.34
Absent	49 43)	95 [82-99]		79 [65-88]		87 [74-94]
PI3K Pathway║
Present	18 (16)	81 [52-93]	0.054	72 [46-87]	0.19	77 [49-91]	0.034
Absent	97 (84)	94 [86-97]		84 [75-90]		93 [85-96]
Ras Pathway
Present	13 (11)	90 [84-96]	0.73	69 [37-87]	0.15	85 [51-96]	0.43
Absent	103 (89)	92 [84-96]		84 [75-90]		91 [83-95]

^*DFS calculated for those who achieved CR and had evaluable MRD for outcome assessment by end-induction MRD or who achieved CR and had evaluable NGS for outcome assessment by genetic alterations

^†15 subjects did not achieve a CR and were not randomized; one participant withdrew from study therapy after achievement of CR, but before randomization

^‡N=115 participants for whom diagnostic samples was available for NGS

^§Includes NOTCH1 and FBXW7 mutations

^║Includes PTEN, AKT, PIK3R1

^¶Includes KRAS, NRAS, NF1, and PTPN11