Phase II Study of Ponatinib in Advanced Gastrointestinal Stromal Tumors: Efficacy, Safety, and Impact of Liquid Biopsy and Other Biomarkers.

ABSTRACT:

Purpose

The purpose of this study is to evaluate ponatinib for advanced gastrointestinal stromal tumors (GIST).

Patients and methods

This single-arm phase II trial enrolled patients with metastatic and/or unresectable GIST with failure of prior tyrosine kinase inhibitor (TKI) treatment into two cohorts based on presence or absence of KIT exon 11 (ex11) primary mutations. Patients initially received ponatinib 45 mg once daily. Following a temporary clinical hold in October 2013, dose reductions were implemented to reduce risk of arterial occlusive events (AOE). Primary endpoint was 16-week clinical benefit rate (CBR) in KIT ex11-positive cohort. KIT mutations in circulating tumor DNA (ctDNA) were assessed.

Results

Forty-five patients enrolled (30 KIT ex11-positive and

SUBMITTER: George S 

PROVIDER: S-EPMC9531534 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature