Table 2.

Molecular predictors of response

GenotypeNo. of patientsCR rate, %UnivariableMultivariableORR rate, %UnivariableMultivariable
POR (95% CI)PPOR (95% CI)P
Total cohort 247 15    42.1    
NGS result   .4    .22   
 No mutation 28 21.4    53.6    
 ≥1 mutation 213 14.6    40.8    
TET2   .09 2.80 (1.09-7.24) .03*  .08 1.92 (0.93-3.94) .07 
 Mut 41 24.3    56.1    
 WT 188 12.8    40.4    
ASXL1   .02* 0.13 (0.02-1.08) .06  .07 0.49 (0.22-1.08) .08 
 Mut 47 4.3    29.8    
 WT 200 17.5    45.0    
TET2-mut/ASXL1-WT   .004* 3.15 (1.20-8.30) .02*  .03* 1.99 (0.86-4.59) .10 
 Present 29 34.5    62.1    
 Other 206 12.1    39.8    
DNMT3A   .31    .85   
 Mut 35 8.6    45.7    
 WT 193 16.1    43.0    
EZH2   >.99    .59   
 Mut 15 13.3    33.3    
 WT 232 15.1    42.7    
DNA methylation mutation   .7    .33   
 Mut 79 16.5    48.1    
 WT 148 14.2    41.2    
Epigenetic regulation   .47    .36   
 Mut 120 13.3    40    
 WT 115 17.4    46.1    
SF3B1   .7    .4   
 Mut 13 7.7    30.8    
 WT 217 15.7    44.2    
Any spliceosome   .048* 0.46 (0.16-1.33) .15  .57 0.86 (0.46-1.63) .65 
 Mut 77 7.8    40.3    
 WT 152 18.4    44.7    
RUNX1   >.99    >.99   
 Mut 24 12.5    41.7    
 WT 223 15.2    42.2    
Signaling pathway   .18    .57   
 Mut 32 6.3    37.5    
 WT 196 16.3    44.4    
TP53   .08 1.29 (0.56-3.0) .55  .79 1.01 (0.54-1.89) .96 
 Mut 82 20.7    43.9    
 WT 165 12.1    41.2    
GenotypeNo. of patientsCR rate, %UnivariableMultivariableORR rate, %UnivariableMultivariable
POR (95% CI)PPOR (95% CI)P
Total cohort 247 15    42.1    
NGS result   .4    .22   
 No mutation 28 21.4    53.6    
 ≥1 mutation 213 14.6    40.8    
TET2   .09 2.80 (1.09-7.24) .03*  .08 1.92 (0.93-3.94) .07 
 Mut 41 24.3    56.1    
 WT 188 12.8    40.4    
ASXL1   .02* 0.13 (0.02-1.08) .06  .07 0.49 (0.22-1.08) .08 
 Mut 47 4.3    29.8    
 WT 200 17.5    45.0    
TET2-mut/ASXL1-WT   .004* 3.15 (1.20-8.30) .02*  .03* 1.99 (0.86-4.59) .10 
 Present 29 34.5    62.1    
 Other 206 12.1    39.8    
DNMT3A   .31    .85   
 Mut 35 8.6    45.7    
 WT 193 16.1    43.0    
EZH2   >.99    .59   
 Mut 15 13.3    33.3    
 WT 232 15.1    42.7    
DNA methylation mutation   .7    .33   
 Mut 79 16.5    48.1    
 WT 148 14.2    41.2    
Epigenetic regulation   .47    .36   
 Mut 120 13.3    40    
 WT 115 17.4    46.1    
SF3B1   .7    .4   
 Mut 13 7.7    30.8    
 WT 217 15.7    44.2    
Any spliceosome   .048* 0.46 (0.16-1.33) .15  .57 0.86 (0.46-1.63) .65 
 Mut 77 7.8    40.3    
 WT 152 18.4    44.7    
RUNX1   >.99    >.99   
 Mut 24 12.5    41.7    
 WT 223 15.2    42.2    
Signaling pathway   .18    .57   
 Mut 32 6.3    37.5    
 WT 196 16.3    44.4    
TP53   .08 1.29 (0.56-3.0) .55  .79 1.01 (0.54-1.89) .96 
 Mut 82 20.7    43.9    
 WT 165 12.1    41.2    

Response rates among the total cohort stratified by genotype; multivariable analysis was performed using a binary logistic-regression model (ORR defined as CR + mCR + PR + HI).

CI, confidence interval; OR, odds ratio.

*

Denotes statistical significance (P < .05).

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