PT - JOURNAL ARTICLE AU - Roman Groisberg AU - David S. Hong AU - Amini Behrang AU - Kenneth Hess AU - Filip Janku AU - Sarina Piha-Paul AU - Aung Naing AU - Siqing Fu AU - Robert Benjamin AU - Shreyaskumar Patel AU - Neeta Somaiah AU - Anthony Conley AU - Funda Meric-Bernstam AU - Vivek Subbiah TI - Characteristics and outcomes of patients with advanced sarcoma enrolled in early phase immunotherapy trials AID - 10.1186/s40425-017-0301-y DP - 2017 Dec 01 TA - Journal for ImmunoTherapy of Cancer PG - 100 VI - 5 IP - 1 4099 - http://jitc.bmj.com/content/5/1/100.short 4100 - http://jitc.bmj.com/content/5/1/100.full SO - J Immunother Cancer2017 Dec 01; 5 AB - Background Immunotherapies, specifically those based on immune checkpoint inhibitors, have shown promising activity in multiple tumor types. Other than mifamurtide (MEPACT®) for osteosarcoma approved by European Medicines Agency, there are no approved immunotherapies for sarcomas.Methods We analyzed medical records of patients with advanced sarcoma who were referred to Phase 1 clinic at MD Anderson and received an immunotherapy (checkpoint inhibitors, vaccines, or cytokine based therapies). Clinical parameters including demographics, clinical history, toxicity, and response were abstracted.Results Among 50 patients enrolled in immunotherapy trials (Bone 10; Soft-tissue 40) we found 14 different subtypes of sarcomas. Royal Marsden Hospital (RMH) prognostic score was <2 (86%). Performance status (PS) was 0–1 in 48 patients (96%); median number of prior therapies was 3 (0–12). Immunotherapy consisted of checkpoint inhibitors (82%: PD1 = 7, PD-L1 = 11, CTLA4 = 22, other = 1) of which 42% were combinations, as well as vaccines (14%), and cytokines (4%). Median overall survival (OS) was 13.4 months (11.2 months: not reached). Median progression free survival (PFS) was 2.4 months (95% CI = 1.9–3.2 months). Best response was partial response (PR) in 2 patients with alveolar soft part sarcoma (ASPS) and stable disease (SD) in 11 patients (3 GIST, 3 liposarcomas (2 DDLS, 1 WDLS), 2 ASPS, 2 leiomyo, 1 osteo). PFS was 34% (23%, at 50%) at 3 months, 16% (8%, 30%) at 6 months, and 6% (2%, 20%) at 1 year. Pseudo-progression followed by stable disease was observed in 2 patients (4%). Grade 3/4 adverse events included rash (10%), fever (6%), fatigue (6%), and nausea/vomiting (6%).Conclusion Immunotherapies were well tolerated in advanced sarcoma patients enrolled in trials. All four ASPS patients had clinical benefit with checkpoint inhibitors and this was the only subtype experiencing partial response. Further evaluation of checkpoint inhibitors in ASPS is warranted.Abbreviations:ASPSAlveolar soft part sarcomaCRComplete responseCTCAECommon Terminology Criteria for Adverse EventsCTLA-4Cytotoxic T-lymphocyte-associated protein 4DDLSDedifferentiated liposarcomaGISTQastrointestinal stromal tumorIRBIInstitutional review boardirRCImmune-related response criteriaLMSLeiomyosarcomaOSOverall survivalPD1Programmed cell death proteinPD-L1Programmed death ligand −1PFSProgression-free survivalPRPartial responsePSPerformance StatusRECISTResponse Evaluation Criteria in Solid TumorsRMHRoyal Marsden HospitalSDStable diseaseTILTumor infiltrating lymphocytesWDLSWell-differentiated liposarcoma