Indication

Platelet-derived growth factor receptor alpha exon 18 mutation(+) gastrointestinal stromal tumor

Platelet-derived growth factor receptor alpha exon 18 mutation(+) gastrointestinal stromal tumor is a molecularly defined subtype of gastrointestinal stromal tumor (GIST), a rare mesenchymal malignancy that typically originates from the interstitial cells of Cajal within the digestive tract, most commonly in the stomach. Read more about Platelet-derived growth factor receptor alpha exon 18 mutation(+) gastrointestinal stromal tumor

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Common Platelet-derived growth factor receptor alpha exon 18 mutation(+) gastrointestinal stromal tumor therapies.

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AYVAKIT (avapritinib)

ANTINEOPLASTIC - PROTEIN-TYROSINE KINASE INHIBITORS·AGGRESSIVE SYSTEMIC MASTOCYTOSIS·+3
BLUEPRINT MEDICINES
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About Platelet-derived growth factor receptor alpha exon 18 mutation(+) gastrointestinal stromal tumor.

Platelet-derived growth factor receptor alpha exon 18 mutation(+) gastrointestinal stromal tumor is a molecularly defined subtype of gastrointestinal stromal tumor (GIST), a rare mesenchymal malignancy that typically originates from the interstitial cells of Cajal within the digestive tract, most commonly in the stomach.

It is characterized by activating oncogenic alterations in exon 18 of the PDGFRA gene—most frequently the D842V point mutation—which cause ligand-independent constitutive kinase signaling driving tumor proliferation.

Clinically, this biomarker-defined entity represents an important therapeutic subgroup because exon 18 activation-loop variants, particularly D842V, confer primary resistance to standard first-generation type II tyrosine kinase inhibitors such as imatinib, necessitating the use of specialized kinase inhibitors targeting this specific mutation.

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