Table 4.
Sarcomas experiencing clinical benefit from a molecularly matched agent with characteristics of their agent and matching.
| MDACC Phase 1 population | AACR GENIE v11.0 population | ||||||||
|---|---|---|---|---|---|---|---|---|---|
| Tumor | Best response | Drug mechanism | Molecular selection | Number with mutation | Total samples | Percentage with mutation | Number with mutation | Total samples | Percentage with mutation |
| Chordoma | PR | MDM2 antagonist | ATM C430S | 1 | 10 | 10% | 2 | 82 | 2.4% |
| Clear cell sarcoma | PR | CMET inhibitor | EWSR1 fusion | 6 | 10 | 60% | 26 | 27 | 96% |
| Clear cell sarcoma | PR | CMET inhibitor | cMET amplification | 2 | 10 | 20% | 0 | 32 | 0% |
| Epithelioid sarcoma | PR | EZH2/EED inhibitor | INI/SMARCB1 loss | 1 | 4 | 25% | 20 | 43 | 47% |
| Epithelioid hemangioendothelioma | PR | VEGF inhibitor + mTOR | TSC1 | 1 | 2 | 50% | 2 | 54 | 4% |
| Inflammatory myofibroblastic tumor | PR | ALK inhibitor | ALK fusion | 2 | 2 | 100% | 17 | 41 | 42% |
| NTRK-rearranged sarcoma | PR, CR | NTRK inhibitor | ETV6-NTRK3,NTRK3-EML4 | 2 | 587 | 0.3% | 27 | 4,036 | 0.66% |
| Clear cell sarcoma | SD > 6 m | MET/VEGFR2 inhibitor | EWSR fusion | 6 | 10 | 60% | 29 | 45 | 64% |
| Clear cell sarcoma | SD > 6 m | EGFR inhbitor | ERBB4 M1017T | 1 | 10 | 10% | 1 | 45 | 2% |
| Low-grade fibromyxoid sarcoma | SD > 6 m | CDK4/6 inhibitor | CDKN2A amplification | 1 | 1 | 100% | 1 | 16 | 6% |
| Sclerosing epithelioid fibrosarcoma | SD > 6 m | SHH inhibitor | PTCH1 | 1 | 9 | 11% | 2 | 19 | 11% |