Table 1:
Patient | Age (years) | Sex | Performance score | PAX-FOXO1 Fusion status | Disease sites at enrollment | Prior courses of XRT | Prior # systemic therapies | Dose level |
---|---|---|---|---|---|---|---|---|
1 | 8 | Female | 70 | FN | Lung, pleura, abdomen, pelvis | 2 | 4 | 1 |
2 | 12 | Female | 70 | FN | Lung, pancreas, pelvis | 2 | 4 | 1 |
3 | 19 | Male | 90 | FP | Lung, mediastinal and hilar LN | 1 | 3 | 1 |
4 | 8 | Female | 100 | FN | Head and neck (temporal fossa)$ | 1 | 4 | 1 |
5^ | 19 | Female | 90 | FP | Lung, pleura, mediastinal and hilar LN | 2 | 2 | 1 |
6 | 11 | Female | 90 | FP | Mediastinum, retroperitoneum, pelvis, bone | 5 | 2 | 1 |
7 | 19 | Female | 90 | FN | Retroperitoneum (paraaortic LN) | 1 | 1 | 1 |
8^ | 8 | Male | 90 | FN | Head and neck (parapharyngeal$ | 1 | 4 | 2 |
9 | 13 | Male | 100 | FP | Lung, pleura, mediastinum | 2 | 3 | 2 |
10 | 18 | Male | 100 | FN | Pleura, mediastinum | 1 | 1 | 2 |
11 | 29 | Male | 80 | FP | Pleura, lung, bone, orbit | 2 | 6 | 2 |
12 | 28 | Male | 80 | FP | Pleura, mediastinum, bone marrow, muscle, pancreas, retroperitoneum, subcutaneous | 2 | 3 | 2 |
13 | 24 | Female | 80 | FP | Lung, pelvis | 2 | 5 | 2 |
Summary* | 18 (8–29) | 6 Male/ 7 Female | 90 (70–100) | 6 FN ERMS/ 7 FP ARMS | 2 Localized$/ 11 Metastatic | 2 (1–5) | 3 (1–6) | 7 DL1/ 6 DL2 |
Not evaluable for toxicity;
Median (range) reported for age, performance score, prior courses of XRT, and prior # systemic therapies;
Local relapse of primary tumor. Abbreviations: XRT = radiation, FN = fusion negative, FP = fusion positive; DL= dose level