American Association for Cancer Research

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Supplementary Figure S1 from Impact of Biomarker-Matched Therapies on Outcomes in Patients with Sarcoma Enrolled in Early-Phase Clinical Trials (SAMBA 101)

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posted on 2023-05-01, 08:20 authored by Roberto Carmagnani Pestana, Justin T. Moyers, Jason Roszik, Shiraj Sen, David S. Hong, Aung Naing, Cynthia E. Herzog, Siqing Fu, Sarina A. Piha-Paul, Jordi Rodon, Timothy A. Yap, Daniel D. Karp, Apostolia M. Tsimberidou, Shubham Pant, Maria A. Zarzour, Ravin Ratan, Vinod Ravi, Robert S. Benjamin, Alexander J. Lazar, Wei-Lien Wang, Najat Daw, Jonathan B. Gill, Douglas J. Harrison, Valerae O. Lewis, Christina L. Roland, Shreyaskumar R. Patel, J. Andrew Livingston, Neeta Somaiah, Joseph A. Ludwig, Anthony P. Conley, Nelson Hamerschlak, Richard Gorlick, Funda Meric-Bernstam, Vivek Subbiah

Treemap of most common drug mechanisms for patients treated in biomarker-matched studies.


National Cancer Institute (NCI)

United States Department of Health and Human Services

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Sheikh Khalifa Bin Zayed Al Nahyan Institute for Personalized Cancer Therapy (Khalifa Institute)



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. 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. 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 (P < 0.001); the clinical benefit rate at 6 months was 43.9% vs. 19.9% (P < 0.001). Progression-free survival was longer for patients in matched trials (median, 5.5 vs. 2.4 months; P < 0.001), and overall survival was also superior for patients in matched trials (median, 21.5 vs. 12.3 months; P < 0.001). The benefit of enrollment in matched trials was maintained when patients with GIST were excluded from the analysis. 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.

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