TABLE 1.
Summary of statistical analysis of in vivo studies
| Models | Rova-T dose | N | Na | KM Med (days) | EFS T − C (days) | EFS T/C | P | minRTV Mean SD (cm3)* | Median response | FPKM | Modified H score |
|---|---|---|---|---|---|---|---|---|---|---|---|
| COG-N-452x | 0.6 mg/kg | 9 | 9 | >42.0 | >32.8 | >4.55 | P < 0.001 | 0.087 ± 0.121 | CR | 9.8 | 190.0 |
| 0.3 mg/kg | 9 | 9 | 37.8 | 28.5 | 4.09 | P < 0.001 | 1.014 ± 0.626 | PD2 | |||
| Felix-PDX | 0.6 mg/kg | 10 | 10 | >41 | >33.5 | >5.45 | P < 0.001 | 0.145 ± 0.121 | PR | 9.3 | 292.5 |
| COG-N-519x | 0.6 mg/kg | 10 | 10 | 33.4 | 25.9 | 4.46 | P < 0.001 | 0.696 ± 0.833 | SD | 4.9 | 300.0 |
| 0.3 mg/kg | 10 | 10 | 15.8 | 8.3 | 2.11 | P < 0.001 | 2.003 ± 0.697 | PD2 | |||
| COG-N-415x | 0.6 mg/kg | 10 | 10 | 20.6 | 12.3 | 2.49 | P = 0.001 | 0.973 ± 0.840 | PD2 | 16.2 | 230.0 |
| 0.3 mg/kg | 10 | 10 | 9.5 | 1.2 | 1.15 | P = 0.370 | 1.619 ± 0.756 | PD1 | |||
| COG-N-415x | 1 mg/kg | 8 | 8 | 42.3 | 37.2 | 8.19 | P = 0.015 | 1.027 ± 1.685 | PR | 16.2 | 230.0 |
| 1 mg/kg (x3) | 8 | 8 | 61.5 | 56.3 | 11.89 | P < 0.001 | 0.159 ± 0.430 | CR | |||
| COG-N-424x | 0.6 mg/kg | 2 | 2 | 22.7 | 18.7 | 5.64 | P = 0.102 | 0.747 ± 0.837 | SD | 19.5 | 255.0 |
| SH-SY5Y | 0.6 mg/kg | 2 | 2 | >36 | >31.5 | >8.08 | P = 0.157 | 0.363 ± 0.514 | SD | N/A | N/A |
| COG-N-421x | 0.6 mg/kg | 2 | 2 | >20 | >13.3 | >2.97 | P = 0.102 | 0.468 ± 0.094 | SD | 8.2 | 182.5 |
| 0.1 mg/kg | 2 | 2 | 8 | 1.3 | 1.19 | P = 1.000 | 2.825 ± 0.676 | PD1 | |||
| KWK-6062x | 0.6 mg/kg | 2 | 2 | 38.4 | 15.1 | 1.65 | P = 0.102 | 0.581 ± 0.138 | PD2 | N/A | 11.3 |
| 0.1 mg/kg | 2 | 2 | 25.5 | 2.2 | 1.09 | P = 0.102 | 0.850 ± 0.224 | PD1 | |||
| SK-N-AS | 0.6 mg/kg | 2 | 2 | 11.6 | −2.1 | 0.85 | P = 1.000 | 0.943 ± 0.781 | PD2 | N/A | 0.0 |
| 0.1 mg/kg | 2 | 2 | 14.3 | 0.6 | 1.04 | P = 1.000 | 1.298 ± 0.609 | PD1 | |||
| NB-FLY-623m | 0.6 mg/kg | 2 | 2 | >28 | >13 | >1.87 | P = 0.102 | 0.804 ± 0.286 | PD1 | 6.1 | 37.5 |
| 0.1 mg/kg | 2 | 2 | 18.2 | 3.2 | 1.21 | P = 0.450 | 1.225 ± 0.420 | PD1 |
Abbreviations: CR, complete response, disappearance of measurable tumor mass during study period; EFS T − C, difference in median time-to-event (days) between T and C groups; EFS T/C, ratio of median time-to-event (days) between T and C groups; KM Med, Kaplan–Meier estimate of median time-to-event (days); Median response, median response evaluation.; N, total number of mice entering experiment; Na, number of mice in analysis; PD, progressive disease, <50% tumor regression throughout study and >25% tumor growth at end of study; PD1, when PD (progressive disease) and the mouse's time to event ≤200% the Kaplan–Meier median time-to-event in control group; PD2, when PD but, additionally, time-to-event is > 200% of the Kaplan-Meier median time-to-event in control group; PR, partial response, ≥50% tumor regression at any point during study but measurable tumor throughout study period.
* minRTV Mean values shown with standard deviations.