Table 1.
Available clinical data on pediatric/adult BRAF-mutant glioblastoma treated with B-Raf-inhibitors.
| First Author | Journal and Year | Study Type | CNS Tumor Type | N. Patients | Drug | Best Response | Treatment Duration/PFS | Final Outcome/OS |
|---|---|---|---|---|---|---|---|---|
| Burger MC | Oncology Reports 2017 | Case Report | V600E GB | 1 | dabrafenib | Nearly CR | 3 months | On treatment |
| Ceccon G | Int J Mol Sci 2018 | Case Report | V600E eGB | 1 | dabrafenib | SD | 10 months | PD |
| Qin C | Front Oncol 2020 | Case Report | V600E GB | 1 | vemurafenib | PR | >2 years | On treatment |
| Robinson GW | BMC Cancer 2014 | Case Report | V600E GB | 1 | vemurafenib | CR | 6 months | On treatment |
| Beba Abadal K | JCO Precision Oncology 2017 | Case Report | V600E GB | 1 | vemurafenib | PR | 11 months | On treatment |
| Meletath SK | JNCCN 2016 | Case Report | V600E HGG (GB/AGG) | 1 | dabrafenib + TTFields | CR | >2 years | On treatment |
| Schreck KC | JNCCN 2018 | Case Report | V600E eGB | 1 | dabrafenib + trametinib | SD | 16 months | On treatment |
| Kushnirsky M | JCO Precision Oncology 2020 | Case Report | V600E GB | 1 | dabrafenib + trametinib | CR | 11 months | On treatment |
| Kanemaru Y | Acta Neuropathol Commun 2019 | Case Report | V600E eGB | 1 | dabrafenib + trametinib | PR | 4 weeks | PD (after treatment suspension for lack of funding) |
| Johanns TM | J Natl Compr Canc Netw 2018 | Case Report | Mixed V600E PXA/eGB | 1 | dabrafenib + trametinib + bevacizumab | PR | Approx. 4 months | PD |
| V600E eGB | 1 | dabrafenib + trametinib | PR | 11 months | PD | |||
| Kaley T | J Clin Oncol 2018 | Basket Trial | V600E Malignant Diffuse Glioma | 11* | vemurafenib | ORR 9.1% (95%CI: 0.2% - 41.3%); CBR 27.3% (95%CI: 6.0% - 61.0%) | mPFS 5.3 months (95% CI: 1.8 - 12.9) | mOS 11.9 months (95% CI: 8.3 - 40.1) |
| Kieran MW | Clin Can Res 2019 | Phase I | V600 HGG and LGG | 10° | dabrafenib | – | 71.7 (5.7–130.4) weeks# | PD |
CR, complete response; PR, partial response; SD, stable disease; PD, progressive disease; CI, confidence interval; mPFS, median progression-free survival; mOS, median overall survival; ORR, overall response rates; CBR, clinical benefit rate; eGB, epithelioid glioblastoma; HGG, high-grade glioma; LGG, low-grade glioma; PXA, anaplastic pleomorphic xanthoastrocytoma; AGG, anaplastic ganglioglioma; GB, glioblastoma; TTFields, Tumor-treating electromagnetic fields. *6 glioblastoma and 5 anaplastic astrocytoma; °2 glioblastomas; #HGG + LGG.