Real-World Outcomes of Oral VEGFR Tyrosine Kinase Inhibitor-Containing Therapy in Unresectable Malignant Mesothelioma: A Single-Center Retrospective Study

Background: Systemic treatment choices after failure of standard therapy are limited for malignant mesothelioma. Oral vascular endothelial growth factor receptor tyrosine kinase inhibitors (VEGFR-TKIs) have been incorporated into salvage treatment strategies, but real-world evidence regarding their effectiveness and safety remains scarce. Methods: We retrospectively analyzed a single-center cohort comprising patients with pathologically confirmed malignant mesothelioma treated at Cancer Hospital, Chinese Academy of Medical Sciences/National Cancer Center between 1 April 2017 and 16 March 2023. Patients with unresectable disease who received a VEGFR-TKI-containing regimen and had sufficient clinical follow-up for outcome assessment were included. Progression-free survival (PFS) served as the primary endpoint. Objective response rate (ORR), disease control rate (DCR), overall survival (OS) from VEGFR-TKI initiation, and recorded adverse events were included as secondary endpoints. Results: The cohort comprised 48 patients, with a median follow-up of 24.0 months. The Kaplan–Meier estimated median PFS was 4.8 months (95% CI, 2.9–8.8), and median OS from VEGFR-TKI initiation was 17.9 months (95% CI, 13.5–not estimable). Among 39 response-evaluable patients, 6 achieved partial response, 20 had stable disease, and 13 had progressive disease as their best overall response, yielding an ORR of 15.4% and a DCR of 66.7%. Two patients remained progression-free for more than 24 months. Grade 3 adverse events in the records included hematuria, hypertension, and nausea/vomiting; no grade 4 or grade 5 adverse event was documented. Conclusions: VEGFR-TKI-containing therapy was associated with clinically relevant disease control in unresectable malignant mesothelioma, with durable benefit observed in a small subset of patients. These observations warrant further evaluation of VEGFR-TKI-based salvage strategies in larger multicenter cohorts.