Data from Impact of Biomarker-Matched Therapies on Outcomes in Patients with Sarcoma Enrolled in Early-Phase Clinical Trials (SAMBA 101)

Abstract

<div>AbstractPurpose:<p>Developing new therapeutics for any of the more than 100 sarcoma subtypes presents a challenge. After progression from standard therapies, patients with sarcoma may be referred for enrollment in early-phase trials. This study aimed to investigate whether enrollment in biomarker-matched early-phase clinical trials leads to better outcomes for patients with advanced sarcoma.</p>Experimental Design:<p>In this retrospective analysis, investigational treatment characteristics and longitudinal survival outcomes were analyzed in patients with biopsy-confirmed sarcoma enrolled in early-phase trials at MD Anderson Cancer Center from May 2006 to July 2021.</p>Results:<p>Five hundred eighty-seven patients were included [405 soft tissue, 122 bone, 60 gastrointestinal stromal tumor (GIST); median of three prior lines of therapy]. Most common subtypes were leiomyosarcoma (17.2%), liposarcoma (14.0%), and GIST (10.2%). Molecular testing was available for 511 patients (87.1%); 221 patients (37.6%) were treated in matched trials. Overall response rate was 13.1% matched compared with 4.9% in unmatched (<i>P</i> < 0.001); the clinical benefit rate at 6 months was 43.9% vs. 19.9% (<i>P</i> < 0.001). Progression-free survival was longer for patients in matched trials (median, 5.5 vs. 2.4 months; <i>P</i> < 0.001), and overall survival was also superior for patients in matched trials (median, 21.5 vs. 12.3 months; <i>P</i> < 0.001). The benefit of enrollment in matched trials was maintained when patients with GIST were excluded from the analysis.</p>Conclusions:<p>Enrollment in biomarker-matched early-phase trials is associated with improved outcomes in heavily pretreated patients with metastatic sarcoma. Molecular testing of tumors from patients with advanced sarcoma and enrollment in matched trials is a reasonable therapeutic strategy.</p></div>