Treatment of Metastatic Gastrointestinal Stromal Tumor with KIT or PDGFRA Mutation

This protocol defines the fourth-line treatment approach for patients with metastatic gastrointestinal stromal tumor (GIST) carrying a KIT or PDGFRA mutation — specifically those without a KIT exon 9 mutation and without a PDGFRA D842V mutation. Mutation genotyping is central to management in this setting.

Clinical Scenario

Metastatic GIST with a confirmed KIT or PDGFRA mutation, excluding the KIT exon 9 and PDGFRA D842V variants. Genotype characterisation is mandatory before treatment selection in advanced and metastatic disease, as it directly determines the appropriate therapy.

Treatment Approach (Partial Overview)

At the fourth-line stage in this genotypically defined population, the protocol specifies a targeted therapy with demonstrated superiority over placebo in a randomised phase III trial. The complete regimen — including full eligibility criteria, monitoring, and sequencing — is available in the structured protocol below.

Instant Access to Structured Evidence-Based Regimens

References

DOI: 10.1177/17588359231192388

Genotype is mandatory for treating advanced/metastatic GIST patients (II,A).
Standard third- and fourth-line treatments are, respectively, regorafenib 160 mg daily 3/1 (I,A) and ripretinib 150 mg once daily (I,A).
The phase III INVICTUS trial proved that ripretinib 150 mg daily was superior to placebo after progression to all standard treatments, showing a PFS of 6.3 months compared to 1.0 month.

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