Table 1.
Demographics and baseline characteristics of patients in the extension cohorts—safety population.
| Extension cohorts (1,000 mg/day) | |||
|---|---|---|---|
| Patients with TGCT | Non-TGCT patientsa | Total | |
| (n = 39) | (n = 52) | (n = 91) | |
| Age, mean (Standard Deviation) (y) | 45.1 (13.99) | 56.2 (14.92) | 51.4 (15.46) |
| Sex, n (%) | |||
| Male | 17 (44%) | 31 (60%) | 48 (53%) |
| Female | 22 (56%) | 21 (40%) | 43 (47%) |
| Race, n (%) | |||
| White | 33 (85%) | 48 (92%) | 81 (89%) |
| Black | 3 (8%) | 1 (2%) | 4 (4%) |
| Asian | 3 (8%) | 1 (2%) | 4 (4%) |
| Native Hawaiian or other Pacific Islander | 0 | 1 (2%) | 1 (1%) |
| Multiple races | 0 | 1 (2%) | 1 (1%) |
| BMI, mean (Standard Deviation) (kg/m2) | 28.03 (5.866) | 26.90 (6.508) | 27.40 (6.225) |
| Tumor location, n | |||
| Knee | 21 | — | — |
| Foot/ankle | 7 | — | — |
| Hip/thigh | 7 | — | — |
| Forearm/wrist | 2 | — | — |
| Elbow | 1 | — | — |
| Gastroc muscle | 1 | — | — |
| Upper extremity | 1 | — | — |
| Previous treatment, n (%) | |||
| Surgery | 31 (80%) | — | — |
| Radiation | 3 (8%) | — | — |
| TKI | 4 (10%) | — | — |
| Other systemic treatment (denosumab or sirolimus) | 2 (5%) | — | — |
Abbreviations: ATC, anaplastic thyroid carcinoma; BMI, body mass index; GIST, gastrointestinal stromal tumor; MEC, mucoepidermal carcinoma; TGCT, tenosynovial giant cell tumor; TKI, tyrosine kinase inhibitor.
aNon-TGCT patients: The 5 non-TGCT cohorts include the following tumor types: ATC (n = 9), GIST (n = 11), malignant effusion (n = 8), MEC (n = 4), and other tumor types (n = 20). Malignant effusions included mesothelioma (2), colon cancer (2), ovarian adenocarcinoma (1), unknown primary with lung and liver metastases (1), breast cancer (1), and non–small cell lung cancer (1). The category of “other tumor types” included mesothelioma (7), malignant peripheral nerve sheath tumor (3), Erdheim–Chester disease (2), neurofibromatosis (2), leiomyosarcoma (1), adenoid cystic carcinoma (1), pancreatic neuroendocrine tumor (1), acinic cell carcinoma of the parotid (1), familial schwannomatosis (1), and desmoplastic small round cell tumor (1).