Table 5 Diagnosis, response, survival, dosing and molecular data of long term survivors
Histological diagnosis | Best response | PFS | OS | Last known status | MTD | Last dose level | Methylation profile | Copy number variations | Mutations and variants |
|---|---|---|---|---|---|---|---|---|---|
Days | Days | mg/m2/d | mg/m2/d | ||||||
High grade glioma | PR | 385 (C) | 1352 (C) | Alive | 280 | 280 | Pilocytic astrocytoma | - | BRAF (p.V600E) |
Medulloblastoma | SD | 422 (C) | 871 (C) | Alive | 580 | 430 | Not attributable; highest score for medulloblastoma group 4 | - | - |
High grade glioma | PR | 441 (NC) | 750 (NC) | Deceased | 530 | 530 | Not attributable; highest score for glioblastoma IDH wt | Homozygous loss of CDKN2A/B; several gains, e.g. 7p (EGFR), 9q (PTCH), 11q (CCND1); several losses, e.g.: 10q (MGMT) | Mutations: TP53 (p.R174X), ATRX (p.R907X), NF1 (p.R1968X); variants (no germline data available): RET (p.R820H), BCRA2 (p.E2856A), ATR (p.Y1462C), PIK3C2G (p.N1211S), TP53 (p.G113S) |
SETTLE tumor | PR | 456 (C) | 1190 (C) | Alive | 580 | 580 | - | No aberrations | Mutations: none; variants: none |
High grade glioma | PR | 499 (C) | 682 (NC) | Deceased | 530 | 280 | - | - | - |