Table 3.
Patient Level Details
| Patient number | Age at Enrollment (Years) | Gender | Molecular Group | Treatment Duration (Months)a | Off Treatment Reason | 12-Week Response Assessment by Central Imaging Review | 12-Week Response Assessment by Enrolling Site | Time to PD or Time to Last Follow-up (Months)a | Survival Status | Time to Death or Time to Last Follow-up (Months)a | Formulation Used |
|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 1.8 | Female | ATRT-MYC | 2.7 | Disease progression | PD | PD | 2.7 | Dead | 3.8 | Liquid |
| 2 | 2.5 | Male | NA | 0.2 | Family decision | – | – | 1.0 | Dead | 1.0 | Liquid |
| 3 | 9.7 | Male | ATRT-MYC | 1.5 | Disease progression | – | – | 1.4 | Dead | 3.1 | ECT |
| 4 | 1.9 | Female | ATRT-SHH | 1.3 | Disease progression | – | – | 1.3 | Dead | 1.4 | Liquid |
| 5 | 1.2 | Male | NA | 1.3 | Disease progression | – | – | 1.3 | Dead | 3.4 | Liquid |
| 6 | 1.9 | Female | ATRT-TYR | 1.3 | Disease progression | – | – | 1.3 | Dead | 52.8 | Liquid |
| 7 | 5.1 | Female | ATRT-SHH | 1.4 | Disease progression | – | – | 1.4 | Dead | 7.6 | ECT |
| 8 | 3.2 | Female | ATRT-TYR | 12.4 | Disease progression | SD | SD | 12.4 | Alive | 73.0 | ECT (1 cycle) & liquid |
| 9 | 2.9 | Male | NA | 1.3 | Disease progression | – | – | 1.3 | Dead | 3.6 | Liquid |
| 10 | 5.9 | Female | ATRT-MYC | 0.1 | Family decision | – | – | 2.0 | Dead | 2.0 | Liquid |
| 11 | 2.1 | Male | ATRT-TYR | 1.3 | Disease progression | – | – | 1.3 | Alive | 53.5 | Liquid |
| 12 | 16.5 | Male | ATRT-MYC | 21.5 | Disease progression | SD | SD | 21.5 | Alive | 66.8 | ECT |
| 13 | 6.2 | Female | ATRT-SHH | 5.1 | Disease progression | SD | SD | 5.1 | Dead | 14.3 | Liquid |
| 14 | 2.3 | Female | ATRT-SHH | 9.0 | Disease progression | PD | SD | 8.9 | Dead | 10.4 | Liquid |
| 15 | 1.9 | Female | ATRT-TYR | 10.9 | Family decision | SD | SD | 61.5 | Alive | 61.5 | ECT (1 cycle) & liquid |
| 16 | 4.4 | Male | NA | 1.4 | Disease progression | – | – | 1.3 | Dead | 3.6 | Liquid |
| 17 | 4.7 | Female | ATRT-SHH | 3.8 | Disease progression | – | – | 3.8 | Dead | 9.1 | Liquid |
| 18 | 4.1 | Female | ATRT-SHH | 5.7 | Disease progression | PR | PR | 5.7 | Dead | 16.4 | ECT (1 cycle) & liquid |
| 19 | 2.3 | Male | ATRT-SHH | 7.0 | Disease progression | SD | SD | 7.0 | Dead | 14.7 | Liquid |
| 20 | 17.7 | Female | ATRT-MYC | 6.7 | Disease progression | SD | SD | 6.6 | Dead | 15.8 | Liquid (1cycle) & ECT |
| 21 | 1.1 | Male | ATRT-MYC | 0.9 | Disease progression | – | – | 0.9 | Dead | 1.3 | Liquid |
| 22 | 11.0 | Female | ATRT-MYC | 6.2 | Physician Request | PD | SD | 15.1 | Dead | 15.1 | ECT |
| 23 | 2.9 | Male | ATRT-MYC | 1.3 | Disease progression | – | – | 1.3 | Dead | 9.4 | Liquid |
| 24 | 1.1 | Male | ATRT-TYR | 1.0 | Disease progression | – | – | 1.0 | Dead | 2.1 | Liquid |
| 25 | 20.2 | Female | ATRT-SHH | 8.8 | Disease progression | SD | SD | 8.8 | Dead | 18.7 | ECT |
| 26 | 5.6 | Female | ATRT-MYC | 1.9 | Disease progression | – | – | 1.9 | Dead | 2.6 | ECT |
| 27 | 2.2 | Female | ATRT-SHH | 1.4 | Disease progression | – | – | 1.4 | Dead | 3.6 | Liquid |
| 28 | 4.0 | Female | ATRT-SHH | 7.6 | Disease progression | SD | SD | 7.4 | Dead | 9.6 | Liquid |
| 29 | 7.9 | Female | ATRT-TYR | 1.3 | Disease progression | – | – | 1.3 | Dead | 2.1 | ECT |
| 30 | 2.8 | Female | ATRT-MYC | 1.4 | Family decision | – | – | 1.9 | Dead | 1.9 | Liquid |
Abbreviations: ECT, enteric-coated tablet; NA, not available; PD, progressive disease; PR, partial response; SD, stable disease.
The two patients who had SD by site assessments but PD by central review are highlighted in green.
aDuration from start of treatment.