Table 3 Effect modifiers of CD19 CAR T cells safety and efficacy.
Subgroup | Overall response (ITT) % (95% CI) | Overall response (mITT) % (95% CI) | Minimal residual disease negative % (95% CI) | Cytokine release syndrome (CRS) % (95% CI) | Grade 3 or higher CRS % (95% CI) | Neurotoxicity % (95% CI) | ||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|
Domain | ||||||||||||
41BB | N = 24 | 78 [72; 83] | N = 27 | 86 [82; 89] | N = 26 | 99 [96; 100] | N = 23 | 86 [76; 92] | N = 25 | 23 [17; 30] | N = 22 | 29 [22; 37] |
CD28 | N = 5 | 58 [51; 63] | N = 5 | 74 [64; 82] | N = 5 | 90 [68; 97] | N = 5 | 83 [75; 89] | N = 5 | 23 [18; 30] | N = 5 | 46 [32; 62] |
p-value | < 0.001* | 0.0059* | 0.0179 | 0.5834 | 0.9713 | 0.0380* | ||||||
Age group | ||||||||||||
Pediatric/young adult | N = 10 | 68 [56; 77] | N = 11 | 82 [72; 89] | N = 11 | 99 [93; 100] | N = 9 | 79 [66; 88] | N = 10 | 22 [14; 33] | N = 8 | 42 [33; 53] |
Mixed | N = 25 | 79 [73; 84] | N = 27 | 86 [83; 89] | N = 25 | 96 [91; 98] | N = 24 | 85 [76; 91] | N = 26 | 20 [15; 27] | N = 22 | 26 [18; 35] |
p-value | 0.048* | 0.3608 | 0.2537 | 0.4555 | 0.7932 | 0.0125* | ||||||
Disease morphology | ||||||||||||
Remission | N = 9 | 84 [80; 87] | N = 10 | 90 [83; 94] | N = 23 | 97 [91; 99] | N = 9 | 82 [61; 93] | N = 10 | 16 [13; 20] | N = 10 | 24 [16; 36] |
Disease | N = 24 | 72 [65; 77] | N = 25 | 83 [78; 86] | N = 10 | 99 [92; 100] | N = 23 | 84 [76; 90] | N = 25 | 23 [17; 30] | N = 18 | 37 [28; 46] |
p-value | < 0.001* | 0.0588 | 0.2831 | 0.8248 | 0.0720 | 0.0948 | ||||||
LD dose | ||||||||||||
High | N = 11 | 66 [56; 75] | N = 11 | 79 [71; 86] | N = 11 | 96 [89; 99] | N = 9 | 80 [69; 88] | N = 11 | 25 [14; 39] | N = 9 | 32 [24; 41] |
Low | N = 17 | 81 [74; 86] | N = 19 | 87 [83; 91] | N = 18 | 98 [94; 100] | N = 17 | 86 [73; 93] | N = 18 | 19 [15; 24] | N = 17 | 32 [21; 45] |
p-value | 0.0093* | 0.0413* | 0.3778 | 0.4142 | 0.3843 | 0.9831 | ||||||
Report type | ||||||||||||
Clinical trial | N = 32 | 76 [70; 81] | N = 35 | 86 [82; 89] | N = 33 | 97 [93; 99] | N = 30 | 85 [78; 90] | N = 33 | 21 [16; 27] | N = 27 | 32 [24; 40] |
RWD | N = 3 | 80 [77; 84] | N = 3 | 83 [80; 87] | N = 3 | 100 [98; 100] | N = 3 | 59 [53; 64] | N = 3 | 18 [15; 22] | N = 3 | 21 [15; 31] |
p-value | 0.1242 | 0.5456 | 0.0377* | 0.0001* | 0.4075 | 0.1061 |