Bevacizumab
- Vascular endothelial growth factor (VEGF) inhibitor: sunitinib
- VHL-haemangioblastoma
- SPS-NF2 Vestibular schwannoma
- Radionecrosis not responsive to steroids
Mammalian target of rapamycin inhibitor (mTOR)
- mTOR inhibitors
- Everolimus
- Indication
- Children >3 yrs and adults
- Recurrent SEGA
- Not surgical resectable
- Bilateral fornix lesions with high risk of morbidity
- Not surgical candidates
- Aim
- Reduce the recurrence rate of SEGAs
- Shrinkage of other tumours associated with tuberous sclerosis (angiofibromas and angiomyolipomas)
- Recurrence of SEGAs after surgery has been found to be low in patients treated with mTOR inhibitors.
- Stabilize tumour
- EXIST 1 trial
- F/U of 28·3 months, 49% patients had a response of > 50% reduction in SEGA volume
- Side effects: stomatitis and pneumonia
- Side effects
- Immunosuppression and recurrent infections,
- Significant anaemia
- Thrombocytopenia
- Fatigue
- Nausea
- Diarrhoea
Vorasidenib
- Indicated for
- IDH1- or IDH2-Mutant Low-Grade Glioma
- Mellinghoff 2023
- Vorasidenib, an oral brain-penetrant inhibitor of mutant IDH1 and IDH2 enzymes
- Inclusion:
- Residual or recurrent grade 2 IDH-mutant glioma
- No previous treatment other than surgery
- Progression-free survival was significantly improved in the vorasidenib group as compared with the placebo group (median progression-free survival,
- 27.7 months vs. 11.1 months: 16 months
- Hazard ratio for disease progression or death, 0.39
BRAF mutation
- Dabrafenib
- Inhibitor of the associated enzyme B-Raf
- Side effects include papilloma (warts), headache, nausea, vomiting, hyperkeratosis (thickening and toughening of the skin), hair loss, rash, joint pain, fever and tiredness
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