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NOTCH1 activating mutations occur in >50% of T-ALL (HD and PEST domains). Gamma-secretase...

Deterministic view of the source YAML entity. Clinical authority remains with the cited source IDs and reviewer sign-off state.

IDBMA-NOTCH1-ACTIVATING-T-ALL
TypeActionability
Statusreviewed 2026-04-27 | pending_clinical_signoff | actionability review required
DiseasesDIS-T-ALL
SourcesSRC-BLAST-GOKBUGET-2018 SRC-CALGB-10403-STOCK-2019 SRC-CIVIC

Actionability Facts

BiomarkerBIO-NOTCH1-MUTATION
Variantactivating (HD or PEST domain)
DiseaseDIS-T-ALL
ESCAT tierIIIA
Recommended combinationsper CALGB-10403 / GMALL / BFM-inspired regimen (1L), nelarabine (R/R T-ALL), venetoclax + navitoclax (early-phase trials)
Evidence summaryNOTCH1 activating mutations occur in >50% of T-ALL (HD and PEST domains). Gamma-secretase inhibitors (GSI) showed activity but were limited by GI toxicity. No GSI is FDA-approved in T-ALL. Standard 1L remains pediatric-inspired regimens (BFM, CALGB-10403). MRD-directed nelarabine/PEG-asparaginase per protocol.

Notes

ESCAT IIIA. OncoKB Level 4. Favorable prognostic in some pediatric-T-ALL series when isolated; adverse with PTEN co-loss.

Used By

No reverse references found in the YAML corpus.