Table 2.
Description of previous treatments, clinically significant mutations and outcomes of patients receiving therapeutic intervention during the study
| Patient ID | General diagnosis | Sample type | Sex | Age (years) | Previous therapy lines | Previous treatment | Previous objective response | Previous PFS (weeks) | Clinical mutations | FPM-guided | Additional intervention | Study treatment | Current objective response | Current PFS (weeks) |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| EV013 | AML | Peripheral blood | M | 5 | 3 | Cyclophosphamide + busulfan + sorafenib | Complete response | 60 | FLT3-ITD, KDM6A | Yes | Allogeneic bone marrow transplant | Fludarabine + cytarabine, midostaurin | Complete response* | 112 |
| EV009 | OS | Excised tumor | F | 9 | 3 | High-dose ifosfamide + larotrectinib | Stable disease | 17 | CDKN2A/B, TP53, ATRX, ZFHX4 | Yes | Resection | Idarubicin + montelukast | Complete response | 84 |
| EV010 | EWS | Excised tumor | M | 21 | 5 | Irinotecan + temozolomide + vigil vaccine | Complete response | 58 | CDKN2A/B, EWSR1-FLI1, ESR2, AD21, TOP2A | Yes | None | Topotecan + cyclophosphamide + vincristine | Complete response | 61 |
| EV002 | AML | Peripheral blood | M | 16 | 2 | Cytarabine | Progressive disease | N/A | CEBPA, ASLX2, SETD2 | Yes | None | Cytarabine + idarubicin + dexamethasone | Complete response | 48 |
| EV004 | RMS | Excised tumor | F | 7 | 3 | Pazopanib + nivolumab | Progressive disease | 2 | CDKN2A/B, GNAC, NRAS | Yes | None | Vincristine, temozolomide + irinotecan | Partial response | 24 |
| EV008 | OS | Excised tumor | F | 17 | 2 | Ifosfamide + mifamurtide | Progressive disease | N/A | CDKN2A/B, TERT, CCDN3 | Yes | Resection | Sirolimus | Stable disease | 15 |
| EV011 | RMS | Excised tumor | M | 4 | 3 | Irinotecan + cyclophosphamide | Progressive disease | N/A | BCOR, PAX3-FOXO1, SMARCA4 | No | None | Cyclophosphamide + vinorelbine + temsirolimus | Partial response | 28 |
| EV021 | NB | Excised tumor | F | 4 | 2 | Temozolomide + irinotecan + dinutuximab | Stable disease | 45 | None | No | None | Cyclophosphamide + topotecan, temozolomide + irinotecan + dinutuximab | Stable disease | 18 |
| EV005 | OS | Biopsy of metastatic nodule | F | 7 | 2 | Ifosfamide | Complete response | 7 | CDKN2A/B, MYC, TP53, 9p24.1, NOTCH3, NF1 | No | None | Sorafenib + everolimus | Progressive disease | N/A |
| EV007 | GBM | Excised tumor | M | 11 | 2 | Bevacizumab + lomustine | Progressive disease | N/A | CREBBP2, TP53, PTEN | No | Radiation | Everolimus + fenofibrate + metformin + celecoxib + etoposide + cyclophosphamide | Progressive disease | N/A |
| EV019 | MB | Excised tumor | F | 12 | 2 | Cisplatin + vincristine + cyclophosphamide | Progressive disease | N/A | TP53, GLI2, MYCN, ATM | No | None | Temozolomide + irinotecan + temsirolimus | Progressive disease | N/A |
| EV022 | AML | Bone marrow aspirate | M | 1.94 | 5 | Clofarabine + cytarabine | Progressive disease | N/A | N/A | No | None | Azacitidine + nivolumab | Progressive disease | N/A |
| EV023 | ALL | Peripheral blood | M | 0.97 | 2 | Vincristine + daunorubicin + cytarabine | Progressive disease | N/A | NRAS, KMT2A-EPS15, EPHA3, PALB2 | No | None | Blinatumomab + methotrexate | Progressive disease | N/A |
| EV025 | RMS | Excised tumor | F | 8 | 2 | Vincristine | Progressive disease | N/A | PAX3-FOXO1 | No | None | Doxorubicin + ifosfamide + mesna | Progressive disease | N/A |
ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; EWS, Ewing sarcoma; GBM, glioblastoma multiforme; MB, medulloblastoma; NB, neuroblastoma; OS, osteosarcoma; RMS, rhabdomyosarcoma. *indicates the patient received a bone marrow transplant.