Table 1.
Baseline demographics and disease characteristics.
| Regorafenib 72 mg/m2 with VI (CON; n = 2) | Regorafenib 72 mg/m2 with VI (SEQ; n = 6) | Regorafenib 82 mg/m2 with VI (SEQ; n = 13) | Total (N = 21) | |
|---|---|---|---|---|
| Male sex, n (%) | 2 (100) | 4 (67) | 7 (54) | 13 (62) |
| Median age, years (range) | 14 (10–17) | 12 (5–15) | 10 (1.5–16) | 10 (1.5–17) |
| Aged <2 years, n (%) | 0 | 0 | 1 (8) | 1 (5) |
| Median BSA, m2 (range) | 1.3 (0.9–1.7) | 1.3 (0.7–1.5) | 1.1 (0.5–1.9) | 1.1 (0.5–1.9) |
| Performance statusa, n (%) | ||||
| 100% | 1 (50) | 3 (50) | 7 (54) | 11 (52) |
| 90% | 0 | 3 (50) | 4 (31) | 7 (33) |
| 70%–80% | 1 (50) | 0 | 2 (15) | 3 (14) |
| Cancer type, n (%) | ||||
| RMS | 1 (50) | 1 (17) | 10 (77) | 12 (57) |
| Alveolar RMS | 0 | 0 | 8 (62) | 8 (38) |
| Embryonal RMS | 1 (50) | 0 | 2 (15) | 3 (14) |
| NOS | 0 | 1 (17) | 0 | 1 (5) |
| Ewing sarcoma | 1 (50) | 4 (67) | 0 | 5 (24) |
| Neuroblastoma | 0 | 1 (17) | 2 (15) | 3 (14) |
| Wilms tumor | 0 | 0 | 1 (8) | 1 (5) |
| Disease status, n (%) | ||||
| Relapsing | 1 (50) | 2 (33) | 10 (77) | 13 (62) |
| Refractory | 1 (50) | 3 (50) | 2 (15) | 6 (29) |
| Relapsing and refractory | 0 | 1 (17) | 1 (8) | 2 (10) |
| Tumor extent at study entry, n (%) | ||||
| Localized disease | 1 (50) | 3 (50) | 3 (23) | 7 (33) |
| Locally advanced | 0 | 1 (17) | 2 (15) | 3 (14) |
| Metastatic disease | 2 (100) | 5 (83) | 9 (69) | 16 (76) |
| Median time in weeks since initial diagnosis (range) | 222 (n = 1) | 80 (42–214; n = 6) | 81 (22–349; n = 12) | 86 (22–349; n = 19) |
| Median time in weeks since most recent progression (range) | 11 (n = 1) | 6 (2–10; n = 6) | 2 (0–13; n = 12) | 3 (0–13; n = 20) |
| Median number of lines of prior therapy | 3.5 | 2.0 | 2.0 | 2.0 |
| Prior anticancer therapies, n (%) | ||||
| Any systemic therapy | 2 (100) | 6 (100) | 13 (100) | 21 (100) |
| Vincristine | 2 (100) | 6 (100) | 13 (100) | 21 (100) |
| Irinotecan | 2 (100) | 2 (33) | 6 (46) | 10 (48) |
| >1 line of systemic treatment | 2 (100) | 5 (83) | 8 (62) | 15 (71) |
| High-dose chemotherapy/ASCR | 0 | 2 (33) | 0 | 2 (10) |
| Any radiotherapy | 2 (100) | 5 (83) | 11 (85) | 18 (86) |
| Any surgical therapeutic procedure | 2 (100) | 4 (67) | 8 (62) | 14 (67) |
Note: Percentages may not total 100% due to rounding.
Abbreviations: ASCR, autologous stem cell rescue; BSA, body surface area; CON, concomitant; NOS, not otherwise specified; RMS, rhabdomyosarcoma; SEQ, sequential; VI, vincristine and irinotecan.
aKarnofsky if aged >12 years; Lansky if aged ≤12 years.