Table 1.
Phase I patient demographics and baseline disease characteristics
| Parameter | Phase I lenvatinib dose-finding cohorta |
|||
|---|---|---|---|---|
| 11 mg/m2 (n = 3) | 14 mg/m2 (n = 9) | 17 mg/m2 (n = 11) | Total (N = 23) | |
| Age, years, median (range) | 12 (3-17) | 15 (5-17) | 12 (6-17) | 12 (3-17) |
| Sex, male, n (%) | 2 (66.7) | 4 (44.4) | 6 (54.5) | 12 (52.2) |
| Lansky play score/Karnofsky performance score, n (%)b | ||||
| 70 | 0 | 1 (11.1) | 1 (9.1) | 2 (8.7) |
| 80 | 0 | 3 (33.3) | 3 (27.3) | 6 (26.1) |
| 90 | 0 | 1 (11.1) | 3 (27.3) | 4 (17.4) |
| 100 | 2 (66.7) | 3 (33.3) | 4 (36.4) | 9 (39.1) |
| Missingc | 1 (33.3) | 1 (11.1) | 0 | 2 (8.7) |
| Classification of solid tumor type, n (%) | ||||
| Rhabdomyosarcoma | 1 (33.3) | 0 | 4 (36.4) | 5 (21.7) |
| Neuroblastoma | 0 | 0 | 3 (27.3) | 3 (13.0) |
| Ewing sarcoma | 0 | 3 (33.3) | 1 (9.1) | 4 (17.4) |
| Osteosarcoma | 1 (33.3) | 0 | 0 | 1 (4.3) |
| Otherd | 1 (33.3) | 6 (66.7) | 3 (27.3) | 10 (43.5) |
| Previous radiotherapy, n (%) | 2 (66.7) | 7 (77.8) | 10 (90.9) | 19 (82.6) |
| Previous systemic therapy, n (%) | 2 (66.7) | 8 (88.9) | 10 (90.9) | 20 (87.0) |
| Chemotherapy | 2 (66.7) | 8 (88.9) | 10 (90.9) | 20 (87.0) |
| Anthracycline | 1 (33.3) | 7 (77.8) | 8 (72.7) | 16 (69.6) |
| Number of prior systemic anticancer therapies, n (%) | ||||
| 0 | 1 (33.3) | 1 (11.1) | 1 (9.1) | 3 (13.0) |
| 1 | 0 | 2 (22.2) | 1 (9.1) | 3 (13.0) |
| 2 | 0 | 0 | 4 (36.4) | 4 (17.4) |
| ≥3 | 2 (66.7) | 6 (66.7) | 5 (45.5) | 13 (56.5) |
| Median Range |
3 0-3 |
3 0-9 |
3 0-6 |
3 0-9 |
| Previous anti-VEGF therapy or other tyrosine kinase inhibitor, n (%) | ||||
| Anti-VEGF therapye | 0 | 2 (22.2) | 0 | 2 (8.7) |
| Other tyrosine kinase inhibitor (afatinib) | 0 | 1 (11.1) | 0 | 1 (4.3) |
Clinical cut-off date: 31 March 2017.
Percentages based on total number of patents within relevant treatment group in the full analysis set.
ECOG PS, Eastern Cooperative Oncology Group performance status; VEGF, vascular endothelial growth factor.
Due to dose capping, four patients in cohort 1 received a lower dose than planned dose level; two were assigned to the lenvatinib 11 mg/m2 group and two were assigned to the lenvatinib 14 mg/m2 group. One additional patient was assigned to the lenvatinib 14 mg/m2 group due to a dose calculation error.
Lansky play scores for patients <16 years of age, Karnofsky performance scores for patients ≥16 years of age.
ECOG PS = 1 for the patient with a planned lenvatinib dose level of 11 mg/m2; ECOG PS = 0 for the patient with a planned lenvatinib dose level of 14 mg/m2.
Includes atypical teratoid rhabdoid tumor (n = 1), atypical teratoid rhabdoid tumor-like (n = 1), alveolar soft part sarcoma (n = 1), anaplastic ependymoma (n = 1), epithelioid sarcoma (n = 1), high-grade glioma (n = 1), high-grade undifferentiated soft tissue sarcoma (n = 1), medulloblastoma (n = 1), papillary thyroid carcinoma (n = 1), and paraganglioma (n = 1).
Bevacizumab (n = 1), sorafenib (n = 1).