Table 2.

Outcomes of NUP98-rearranged hematological malignancy

TumorAgeNo. of patientsFusion partnerOutcomeReference
TotalFusion
AML Pediatric 124 NSD1 33.3% 4-y EFS and OS 59  
AML Pediatric 246 15 NSD1 13%, 52%, 81% 3-y EFS, OS, CIR, respectively 32  
AML Pediatric 181 25 NSD1 25% and 33% EFS with and without FLT3 alteration, respectively 33  
AML Pediatric 98 18 Mostly NSD1 (also KDM5A, TOP130% and 48% 5-y DFS and OS, respectively 31  
AML Pediatric 385 10 9 NSD1, 1 KDM5A 10% and 25% EFS and OS, respectively 60  
AML <3 y 232 KDM5A 33.3%, 16.7%, 83.3% 3-y OS, EFS, CIR, respectively 61  
AEL Pediatric 35 5 KDM5A, 2 NSD1 ∼35% 5-y OS 3  
AMKL Pediatric 87 KDM5A 35%, 25%, 62% 5-y OS, EFS, CIR, respectively 62  
AMKL Pediatric 153 14 KDM5A 36%, 36%, 36% 4-y OS, EFS, CIR, respectively 63  
FLT3-ITD+ AML Pediatric 44 NSD1 4% complete response rate 64  
AML Pediatric 238 12 NSD1 31%, 8%, 83% 4-y OS, EFS, CIR, respectively 34  
AML Adult 727 NSD1 11%, 0%, 89% 4-y OS, EFS, CIR, respectively 34  
AML Pediatric 2381 46 KDM5A 34%, 30%, 64% 4-y OS, EFS, RR, respectively 55  
AML Adult 17 16 HOXA9 4-mo median DFS, 8-mo median OS 65  
AML Adult 493 11 HOXA9 13.5-mo median OS, 6-mo median DFS 66  
AML Adult 257 NSD1 1.8-mo median EFS 67  
AML Younger adult, <60 y 504 NSD1 43% complete response rate 68  
TumorAgeNo. of patientsFusion partnerOutcomeReference
TotalFusion
AML Pediatric 124 NSD1 33.3% 4-y EFS and OS 59  
AML Pediatric 246 15 NSD1 13%, 52%, 81% 3-y EFS, OS, CIR, respectively 32  
AML Pediatric 181 25 NSD1 25% and 33% EFS with and without FLT3 alteration, respectively 33  
AML Pediatric 98 18 Mostly NSD1 (also KDM5A, TOP130% and 48% 5-y DFS and OS, respectively 31  
AML Pediatric 385 10 9 NSD1, 1 KDM5A 10% and 25% EFS and OS, respectively 60  
AML <3 y 232 KDM5A 33.3%, 16.7%, 83.3% 3-y OS, EFS, CIR, respectively 61  
AEL Pediatric 35 5 KDM5A, 2 NSD1 ∼35% 5-y OS 3  
AMKL Pediatric 87 KDM5A 35%, 25%, 62% 5-y OS, EFS, CIR, respectively 62  
AMKL Pediatric 153 14 KDM5A 36%, 36%, 36% 4-y OS, EFS, CIR, respectively 63  
FLT3-ITD+ AML Pediatric 44 NSD1 4% complete response rate 64  
AML Pediatric 238 12 NSD1 31%, 8%, 83% 4-y OS, EFS, CIR, respectively 34  
AML Adult 727 NSD1 11%, 0%, 89% 4-y OS, EFS, CIR, respectively 34  
AML Pediatric 2381 46 KDM5A 34%, 30%, 64% 4-y OS, EFS, RR, respectively 55  
AML Adult 17 16 HOXA9 4-mo median DFS, 8-mo median OS 65  
AML Adult 493 11 HOXA9 13.5-mo median OS, 6-mo median DFS 66  
AML Adult 257 NSD1 1.8-mo median EFS 67  
AML Younger adult, <60 y 504 NSD1 43% complete response rate 68  

AEL, acute erythroid leukemia; AMKL, acute megakaryoblastic leukemia; CIR, cumulative incidence of relapse; DFS, disease-free survival; EFS, event-free survival; OS, overall survival; RR, relapse rate.

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