Table 1. Patients with Ewing sarcoma tested for germline mutations in PTPRD and outcomes with IGF-1R-based therapy.
| Age at diagnosis | Mutation | Time from diagnosis metastasis | Best RECIST response to IGF-1R inhibitor monotherapy (PFS) | Best RECIST response to IGF-1R+mTOR inhibitor (PFS) | |
|---|---|---|---|---|---|
| 1 | 24 | V253I, W775stop | 6 months | CR, −100% (3 years) | CR, −100% (2 years) |
| 2 | 22 | T781A | 0 | Not treated | PD, +21% |
| 3 | 33 | Wild-type | 7 years | Not treated | SD, −27% (16+ months) |
| 4 | 18 | Wild-type | 2 years | Not treated | SD, −23% (20 months) |
| 5 | 13 | R995C | 1 years | Not treated | CR, −100% (28 months) |
| 6 | 21 | Wild-type | 0 | Not treated | PD, −42%, new lesion |
| 7 | 13 | Wild-type | 0 | Not treated | SD, −14% (4.5 months) |
| 8 | 13 | Wild-type | 0 | Not treated | Not treated |
Abbreviations: RECIST, response evaluation criteria in solid tumors; IGF-1R, insulin-like growth factor receptor 1; CR, complete response; PD, progressive disease; SD, stable disease