Table 1.
Demographics and baseline characteristics
| Patients | Cohort A (n = 24) | Cohort B (n = 12) |
|---|---|---|
| Age (years; mean ± SD) | 28 ± 9 | 11 ± 3 |
| Sex, n (%) | ||
| Male | 19 (79.2) | 5 (41.7) |
| Female | 5 (20.8) | 7 (58.3) |
| ECOG performance status at enrollment, n (%) | ||
| 0 | 19 (79.2) | 10 (100.0) |
| 1 | 5 (20.8) | 2 (16.7) |
| Presence of metastasis, n (%) | ||
| No (local advanced disease) | 1 (4.2) | 2 (16.7) |
| Yes | 23 (95.8) | 10 (83.3) |
| Primary tumor location, n (%) | ||
| Extremities | 9 (37.5) | 5 (41.7) |
| Axial skeleton | 6 (25.0) | 3 (25.0) |
| Other | 9 (37.5) | 4 (33.3) |
| Site of target and non‐target lesions, n (%) | ||
| Lung only | 11 (45.8) | 4 (33.3) |
| Other organs involved | 13 (54.2) | 8 (66.7) |
| Time from first diagnosis to enrollment, n (%) | ||
| ≤24 months | 22 (91.7) | 12 (100.0) |
| >24 months | 2 (8.3) | 0 (0.0) |
| Lines of previous chemotherapy, n (%) | ||
| 1 | 15 (62.5) | 8 (66.7) |
| ≥2 | 9 (37.5) | 4 (33.3) |
| EWS‐FLI1 translocation, n (%) | ||
| No | 1 (4.2) | 1 (8.3) |
| Yes | 11 (33.3) | 9 (75.0) |
| Not available | 12 (50.0) | 2 (16.7) |
Abbreviations: ECOG, Eastern Cooperative Oncology Group; EWS, Ewing sarcoma.