Characteristics and outcomes of patients with advanced sarcoma enrolled in early phase immunotherapy trials

作者：Roman Groisberg, David S. Hong, Behrang Amini, Kenneth R. Hess, Filip Jankú, Sarina Anne Piha-Paul, Aung Naing, Siqing Fu, Robert S. Benjamin, SHREYASKUMAR R. PATEL, Neeta Somaiah, Anthony Paul Conley, Funda Meric‐Bernstam, Vivek Subbiah · 发表于：Journal for ImmunoTherapy of Cancer · 年份：2017 · DOI：10.1186/s40425-017-0301-y · 被引用次数：148 · 研究领域：Sarcoma Diagnosis and Treatment、Cancer Immunotherapy and Biomarkers、Immunotherapy and Immune Responses

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% ...