Table 2. Early trial results in patients... | American Society of Hematology

Table 2.

Early trial results in patients with molecular failure after firstline therapy

Patient characteristics	N	Treatment	MRD response	Clinical outcome	Reference
NPM1 MRD failure	10	AZA	7 of 10 molecular response	CR sustained in 7 of 10, with median follow-up of 10 mo	88
RT-qPCR <1% or loss of donor chimerism	53 (32 NPM1mut)	AZA	36% MRDneg	1 y RFS, 46%	89
NPM1 MRD failure	33	20 chemotherapy/HMA ± HCT 13 direct HCT	80% MRDneg (8/10 after chemotherapy only)	2 y OS, 86%	90
NPM1 MRD relapse	30	27 chemotherapy + HCT 3 direct HCT	59% MRDneg (16/27 after chemotherapy only)	2 y OS, 63%	19
NPM1 MRD failure or NPM1 MRD relapse	5 7	VEN + HMA/LDAC	5 of 5 CR MRDneg 6 of 7 CR MRDneg	All responders had MRDneg during median follow-up of 12 mo	91
NPM1 MRD failure or NPM1 MRD relapse	2 9	VEN + AZA + HCT	11 of 11 CR MRDneg (9/11 after VEN + AZA only)	CR sustained in 9/10 with median follow-up of 26 mo	92
MFC-MRD relapse	16	7 HMA-based chemotherapy 9 direct HCT	43% MRDneg (3/7 after chemotherapy only)	5 y RFS, 31% 5 y OS, 45%	93
MRD relapse	26 (20 NPM1mut)	VEN-LDAC	54% MRDneg	2 y EFS, 54% 2 y OS, 73%	94
Molecular MRD failure	19	VEN ± LDAC or HMA or other	84% molecular remission	Median OS, 18.4 mo	95
MRD (NPM1 or other gene fusions) failure with baseline FLT3mut	48 (39 NPM1mut)	32 gilteritinib 8 quizartinib 8 sorafenib	40% MRDneg	2 y OS, 80%	96

Patient characteristics	N	Treatment	MRD response	Clinical outcome	Reference
NPM1 MRD failure	10	AZA	7 of 10 molecular response	CR sustained in 7 of 10, with median follow-up of 10 mo	88
RT-qPCR <1% or loss of donor chimerism	53 (32 NPM1mut)	AZA	36% MRDneg	1 y RFS, 46%	89
NPM1 MRD failure	33	20 chemotherapy/HMA ± HCT 13 direct HCT	80% MRDneg (8/10 after chemotherapy only)	2 y OS, 86%	90
NPM1 MRD relapse	30	27 chemotherapy + HCT 3 direct HCT	59% MRDneg (16/27 after chemotherapy only)	2 y OS, 63%	19
NPM1 MRD failure or NPM1 MRD relapse	5 7	VEN + HMA/LDAC	5 of 5 CR MRDneg 6 of 7 CR MRDneg	All responders had MRDneg during median follow-up of 12 mo	91
NPM1 MRD failure or NPM1 MRD relapse	2 9	VEN + AZA + HCT	11 of 11 CR MRDneg (9/11 after VEN + AZA only)	CR sustained in 9/10 with median follow-up of 26 mo	92
MFC-MRD relapse	16	7 HMA-based chemotherapy 9 direct HCT	43% MRDneg (3/7 after chemotherapy only)	5 y RFS, 31% 5 y OS, 45%	93
MRD relapse	26 (20 NPM1mut)	VEN-LDAC	54% MRDneg	2 y EFS, 54% 2 y OS, 73%	94
Molecular MRD failure	19	VEN ± LDAC or HMA or other	84% molecular remission	Median OS, 18.4 mo	95
MRD (NPM1 or other gene fusions) failure with baseline FLT3mut	48 (39 NPM1mut)	32 gilteritinib 8 quizartinib 8 sorafenib	40% MRDneg	2 y OS, 80%	96

LDAC, low-dose cytarabine; MRDneg, negative MRD status; VEN, venetoclax.