• Alice P Chen, Elad Sharon, Geraldine O'Sullivan-Coyne, Nancy Moore, Jared C Foster, James S Hu, Brian A Van Tine, Anthony P Conley, William L Read, Richard F Riedel, Melissa A Burgess, John Glod, Elizabeth J Davis, Priscilla Merriam, Abdul R Naqash, Kristin K Fino, Brandon L Miller, Deborah F Wilsker, Asma Begum, Katherine V Ferry-Galow, Hari A Deshpande, Gary K Schwartz, Brian H Ladle, Scott H Okuno, Jill C Beck, James L Chen, Naoko Takebe, Laura K Fogli, Christina L Rosenberger, Ralph E Parchment, and James H Doroshow.
• From the Division of Cancer Treatment and Diagnosis (A.P. Chen, E.S., G.O.-C., N.M., J.C.F., A.R.N., N.T., L.K.F., C.L.R., J.H.D.) and the Center for Cancer Research (J.G., J.H.D.), National Cancer Institute, Bethesda, the Clinical Pharmacodynamics Biomarker Program, Applied and Developmental Research Directorate, Frederick National Laboratory for Cancer Research, Frederick (K.K.F., B.L.M., D.F.W., A.B., K.V.F.-G., R.E.P.), and the Department of Oncology, Johns Hopkins University School of Medicine and Sidney Kimmel Comprehensive Cancer Center, Baltimore (B.H.L.) - all in Maryland; the Division of Oncology, University of Southern California Norris Comprehensive Cancer Center, Los Angeles (J.S.H.); the Division of Oncology, Washington University School of Medicine in St. Louis, St. Louis (B.A.V.T.); the University of Texas M.D. Anderson Cancer Center, Houston (A.P. Conley); Emory University, Atlanta (W.L.R.); Duke Cancer Institute, Duke University Medical Center, Durham, NC (R.F.R.); University of Pittsburgh Medical Center Hillman Cancer Center, Pittsburgh (M.A.B.); the Division of Hematology-Oncology, Department of Medicine, Vanderbilt University Medical Center, Nashville (E.J.D.); the Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston (P.M.); the Department of Internal Medicine, Section of Medical Oncology, Yale University School of Medicine, New Haven, CT (H.A.D.); the Division of Hematology and Oncology, Department of Medicine, Columbia University Irving Medical Center, New York (G.K.S.); Mayo Clinic, Rochester, MN (S.H.O.); the Division of Pediatric Hematology-Oncology, University of Nebraska Medical Center, Omaha (J.C.B.); the Division of Medical Oncology, Department of Internal Medicine, Ohio State University, Columbus (J.L.C.); and Stephenson Cancer Center at the University of Oklahoma, Oklahoma City (A.R.N.).
• N. Engl. J. Med. 2023 Sep 7; 389 (10): 911921911-921.

BackgroundAlveolar soft part sarcoma (ASPS) is a rare soft-tissue sarcoma with a poor prognosis and no established therapy. Recently, encouraging responses to immune checkpoint inhibitors have been reported.MethodsWe conducted an investigator-initiated, multicenter, single-group, phase 2 study of the anti-programmed death ligand 1 (PD-L1) agent atezolizumab in adult and pediatric patients with advanced ASPS. Atezolizumab was administered intravenously at a dose of 1200 mg (in patients ≥18 years of age) or 15 mg per kilogram of body weight with a 1200-mg cap (in patients <18 years of age) once every 21 days. Study end points included objective response, duration of response, and progression-free survival according to Response Evaluation Criteria in Solid Tumors (RECIST), version 1.1, as well as pharmacodynamic biomarkers of multistep drug action.ResultsA total of 52 patients were evaluated. An objective response was observed in 19 of 52 patients (37%), with 1 complete response and 18 partial responses. The median time to response was 3.6 months (range, 2.1 to 19.1), the median duration of response was 24.7 months (range, 4.1 to 55.8), and the median progression-free survival was 20.8 months. Seven patients took a treatment break after 2 years of treatment, and their responses were maintained through the data-cutoff date. No treatment-related grade 4 or 5 adverse events were recorded. Responses were noted despite variable baseline expression of programmed death 1 and PD-L1.ConclusionsAtezolizumab was effective at inducing sustained responses in approximately one third of patients with advanced ASPS. (Funded by the National Cancer Institute and others; ClinicalTrials.gov number, NCT03141684.).Copyright © 2023 Massachusetts Medical Society.

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