Ripretinib could become a potential therapeutic option for gastrointestinal stromal tumor (GIST), according to a study recently published in Advances in Therapy.

“Ripretinib showed a clinically meaningful benefit and a tolerable safety profile as a fourth- or later-line therapy in patients with advanced GIST in the real-world setting,” the authors wrote.

This prospective, large-scale, real-world registry study included 240 patients previously diagnosed with GIST from the Patient Assistance Program (PAP).

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All participants had previously received 3 or more lines of tyrosine kinase inhibitors and had also undergone treatment with ripretinib for at least 2 cycles. The age range varied from 21.6 to 90.7 years, with a median of 58.1 years, and most (66.6%) patients were male. One-third of the patients (33.3%) had a lesion with a diameter greater than 10 cm, and a similar proportion (37.5%) had an Eastern Cooperative Oncology Group (ECOG) performance status score of 2 or more points. The tumor appeared most frequently in the small intestine (58.7%), and almost all (94.5%) were metastatic. The most common mutation was KIT 11, present in 49.6% of the patients.

The median progression-free survival was 7.7 months after a median follow-up of 6.5 months. The subsequent multivariate analysis determined that the ECOG score correlated with progression-free survival. Benefits were especially noted in cases of nongastric GISTs.

Moreover, the tumor shrinkage rate was 43%, while the disease control rate was 73%. The effects of ripretinib occurred among cases with different gene mutations.

Regarding the safety profile, the most common adverse event was alopecia, present in 17.1% of the patients, followed closely by hand-foot syndrome in 15.4%.

Furthermore, the visual analog score increased among patients treated with ripretinib, with an average increase of 3.7 points. In comparison, those who did not receive this drug had an average decline of 8.9 points.

“The assessment of [quality of life] in the real-world setting is valuable for late-line use in more fragile patients,” the authors wrote.


Zhang X, Zhang P, Qiu H, et al. Large-scale, multicenter, prospective registry study of ripretinib in advanced gist: a real-world study from China. Adv Ther. Published online June 25, 2023. doi:10.1007/s12325-023-02576-0