TABLE 1.
Patient demographics and baseline characteristics
| Characteristic |
Cohort 1 (60 mg/d) (n = 3) |
Cohort 2 (90 mg/d) (n = 11) |
Cohort 3 (120 mg/d) (n = 4) |
Total (N = 18) |
|---|---|---|---|---|
| Age (y) | 57.0 (38‐67) | 50.0 (31‐74) | 45.5 (42‐59) | 49.0 (31‐74) |
| Male, n (%) | 3 (100.0) | 4 (36.4) | 3 (75.0) | 10 (55.6) |
| Body weight (kg) | 67.50 (60.4‐80.0) | 58.50 (44.1‐75.9) | 53.15 (45.2‐70.2) | 59.45 (44.1‐80.0) |
| Previous treatment, n (%) | ||||
| Cancer therapy (excluding radiation and surgery) | 3 (100.0) | 10 (90.9) | 4 (100.0) | 17 (94.4) |
| Cancer surgery | 3 (100.0) | 7 (63.6) | 2 (50.0) | 12 (66.7) |
| Radiation therapy | 0 (0.0) | 4 (36.4) | 2 (50.0) | 6 (33.3) |
| Number of prior cancer drugs, n (%) | ||||
| 0 | 0 (0.0) | 1 (9.1) | 0 (0.0) | 1 (5.6) |
| 1 | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| 2 | 0 (0.0) | 1 (9.1) | 0 (0.0) | 1 (5.6) |
| 3 or more | 3 (100.0) | 9 (81.8) | 4 (100.0) | 16 (88.9) |
| Cancer type, n (%) | ||||
| Sarcoma | 0 (0.0) | 5 (45.5) | 0 (0.0) | 5 (27.8) |
| Dedifferentiated liposarcoma | 0 (0.0) | 2 (18.2) | 0 (0.0) | 2 (11.1) |
| Leiomyosarcoma | 0 (0.0) | 2 (18.2) | 0 (0.0) | 2 (11.1) |
| Unknown | 0 (0.0) | 1 (9.1) | 0 (0.0) | 1 (5.6) |
| Breast | 0 (0.0) | 0 (0.0) | 1 (25.0) | 1 (5.6) |
| Stomach | 0 (0.0) | 1 (9.1) | 0 (0.0) | 1 (5.6) |
| Other | 3 (100.0) | 4 (36.4) | 2 (50.0) | 9 (50.0) |
| Epipharynx | 0 (0.0) | 0 (0.0) | 1 (25.0) | 1 (5.6) |
| Gallbladder | 0 (0.0) | 1 (9.1) | 0 (0.0) | 1 (5.6) |
| Parotid gland | 0 (0.0) | 1 (9.1) | 0 (0.0) | 1 (5.6) |
| Submandibular gland | 1 (33.3) | 1 (9.1) | 0 (0.0) | 2 (11.1) |
| Thymic carcinoma | 1 (33.3) | 1 (9.1) | 0 (0.0) | 2 (11.1) |
| Urachal | 1 (33.3) | 0 (0.0) | 0 (0.0) | 1 (5.6) |
| Vater's Papilla | 0 (0.0) | 0 (0.0) | 1 (25.0) | 1 (5.6) |
| Unknown primary | 0 (0.0) | 1 (9.1) | 1 (25.0) | 2 (11.1) |
| Cancer stage, n (%) | ||||
| II | 0 (0.0) | 0 (0.0) | 1 (25.0) | 1 (5.6) |
| IV | 3 (100.0) | 11 (100.0) | 2 (50.0) | 16 (88.9) |
| Other | 0 (0.0) | 0 (0.0) | 1 (25.0) | 1 (5.6) |
| ECOG PS, n (%) | ||||
| 0 | 3 (100.0) | 9 (81.8) | 2 (50.0) | 14 (77.8) |
| 1 | 0 (0.0) | 2 (18.2) | 2 (50.0) | 4 (22.2) |
| TP53 genotype, n (%) | ||||
| Wild‐type | 1 (33.3) | 7 (63.6) | 3 (75.0) | 11 (61.1) |
| Mutant | 2 (66.7) | 3 (27.3) | 1 (25.0) | 6 (33.3) |
| Indeterminate a | 0 (0.0) | 1 (9.1) | 0 (0.0) | 1 (5.6) |
| Number of prior cancer drugs, n (%) | ||||
| 0 | 0 (0.0) | 1 (9.1) | 0 (0.0) | 1 (5.6) |
| 1 | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| 2 | 0 (0.0) | 1 (9.1) | 0 (0.0) | 1 (5.6) |
| ≥3 | 3 (100.0) | 9 (81.8) | 4 (100.0) | 16 (88.9) |
Values of continuous variables are presented as median (range).
Abbreviation: ECOG PS, Eastern Cooperative Oncology Group Performance Status.
a
Indeterminate with some reasons such as the sample cannot be amplified sufficiently and the allele frequency is below the detection limit.