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Molecular Profile | TSC1 mutant |
Therapy | Everolimus |
Indication/Tumor Type | subependymal giant cell astrocytoma |
Response Type | predicted - sensitive |
Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
---|---|---|---|---|---|---|---|
TSC1 mutant | subependymal giant cell astrocytoma | predicted - sensitive | Everolimus | Phase III | Actionable | In a Phase III trial (EXIST-1), Afinitor (everolimus) treatment resulted in a 50% or more tumor reduction in 35% (27/78) of adult and pediatric patients diagnosed with tuberous sclerosis complex and had subependymal giant cell astrocytoma, compared to 0% (0/39) in the placebo group, 85% (99/117) of the patients harbored mutations in TSC1 and/or TSC2 (PMID: 23158522; NCT00789828). | 23158522 |
PubMed Id | Reference Title | Details |
---|---|---|
(23158522) | Efficacy and safety of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis complex (EXIST-1): a multicentre, randomised, placebo-controlled phase 3 trial. | Full reference... |