Real-world data on immune checkpoint inhibitors in advanced sarcomas across multiple European institutions

BACKGROUND: Following the success of immune checkpoint inhibitors (ICI) in other cancer types, their role is being evaluated in sarcomas. They have been assessed as monotherapy, or in combination with other ICI, chemotherapeutic drugs and tyrosine kinase inhibitors (TKI) in several clinical trials. So far the results have been limited to non-selected sarcoma populations. Further work is required to select patients who will benefit from immunotherapy. PATIENTS AND METHODS: We conducted a pooled retrospective analysis of the use of ICI in patients with advanced sarcomas in multiple European institutions. ICI-based treatments included ICI monotherapy (n = 43, 59.7%), double ICI (n = 5, 6.9%), ICI plus TKI (n = 21, 29.2%) and ICI plus chemotherapy (n = 3, 4.2%). RESULTS: Seventy-two patients from 10 European institutions, with metastatic (87.5%) or locally advanced (12.5%) disease were included. The most common subtype was undifferentiated pleomorphic sarcoma (16.7%), followed by leiomyosarcoma (12%), liposarcoma (10%) and angiosarcoma (9.7%). The median number of prior lines of systemic therapy was 2 (0-8). The objective response rate was 34.4% and was higher in combination regimens versus ICI monotherapy. With a median follow-up of 20.7 months, median progression-free survival (PFS) was 4.6 and median overall survival (OS) 18.8 months. Line of therapy (1st/2nd vs. ≥ 3rd line) and best response to ICI was significantly associated with PFS and OS. Histological subtype was significantly associated with OS. Toxicity was in general manageable; only six (8.3%) patients discontinued therapy for AE. INTERPRETATION: Our study provided additional real-world data on the outcome of ICI in patients with advanced sarcomas.