survival (PFS), overall survival, safety, and EORTC QLQ-C30-based quality of life (QoL) at baseline and 3 months.Results: 16 patients were enrolled (median age 53.3 years; 75% male); median number of prior lines was 3 (range 3-5), and 44% had ECOG PS 2. The most common primary sites were stomach (50%) and small intestine (25%); 81.2% had liver metastases, with a median of 3 disease sites.Primary mutations were KIT exon 11 (56%), exon 9 (25%), and SDH-deficient GIST (12.5%).The majority of secondary mutations were exon 11+ 17(43%).Among 15 response-evaluable patients, the 3-month ORR was 20.0% and 6.6% by Choi and RECIST v1.1, respectively.The 3-month PFR was 53.3% by RECIST v1.1 and 46.0% by Choi, with a median PFS of 4.0 months.The most common grade 3 adverse events were hand-foot skin reaction (20%), diarrhoea (13.3%) and hypertension (20%).Treatment interruptions and dose reductions occurred in 80% and 73.3% of patients, respectively; the median delivered cabozantinib dose was 44 mg.QoL analysis (n=12) showed non-significant worsening in several functional and gastrointestinal symptom domains and in global health status/ QoL. Conclusions:Cabozantinib demonstrated clinically meaningful antitumour activity with a manageable safety profile in heavily pretreated, poor ECOG-PS GIST patients and may represent a therapeutic option where access to ripretinib is limited.Larger phase III studies are required to confirm its efficacy.
Kaparaboyna et al. (Sun,) studied this question.