Avelumab Plus Axitinib Shows Promise in Advanced GIST

Medscape

6 hours ago

Combination therapy with avelumab plus axitinib demonstrated a clinical benefit in patients with advanced gastrointestinal stromal tumour (GIST), achieving a 69.6% disease control rate and a 57.1% progression-free survival (PFS) rate at 3 months.
METHODOLOGY:
Researchers conducted a phase 2 trial to evaluate the safety and efficacy of avelumab in combination with axitinib among 56 patients with unresectable/metastatic GIST (median age, 60 years) after the failure of standard therapy.
Participants received avelumab 10 mg/kg intravenously every 2 weeks combined with axitinib 5 mg orally twice daily.
The primary endpoint was the 3-month PFS rate; key secondary endpoints included overall survival, disease control rate, duration of response, and adverse events (AEs).
The median follow-up duration was 27.4 months.
TAKEAWAY:
The PFS rate at 3 months was 57.1%, the median PFS was 4.6 months, and the median overall survival was 14.2 months.
A partial response was achieved in 8.9% of patients, and the median duration of response was 18.5 months.
Stable disease was observed in 60.7% of patients, corresponding to a disease control rate of 69.6%.
AEs occurred in 94.6% of patients, with 30.4% experiencing grade 3 or higher events. The most common treatment-related AEs were diarrhoea (33.9%), thyroid dysfunction (30.4%), hypertension (25%), palmar-plantar erythrodysesthesia (23.2%), myalgia (17.9%), and dysphonia (17.9%).
IN PRACTICE:
"The AXAGIST trial is the largest prospective study evaluating the efficacy of the combination of immune checkpoint inhibitor and a multikinase inhibitor in patients with advanced/metastatic GIST. These results provide unique data on the activity of axitinib in GIST patients, which in combination with avelumab, resulted in long-term clinical benefit in a significant subset of patients," the authors wrote.
SOURCE:
This study was led by Piotr Lukasz Rutkowski, Maria Skłodowska-Curie National Research Institute of Oncology, Warsaw, Poland. It was published online on June 06, 2025, in the European Journal of Cancer .
LIMITATIONS:
This study lacked a comparator arm, which made it impossible to conclude whether the addition of avelumab improves the antitumour activity of axitinib.
DISCLOSURES:
This research and drug supply were financially supported by Pfizer, as part of an alliance between Merck KGaA, Darmstadt, Germany, and Pfizer Inc. Several authors declared receiving honoraria or having other ties with various sources.