{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,11,11]],"date-time":"2025-11-11T13:36:31Z","timestamp":1762868191131,"version":"3.37.3"},"reference-count":17,"publisher":"Ovid Technologies (Wolters Kluwer Health)","issue":"26","license":[{"start":{"date-parts":[[2020,6,26]],"date-time":"2020-06-26T00:00:00Z","timestamp":1593129600000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0"}],"funder":[{"name":"Bayer SpA","award":["-"],"award-info":[{"award-number":["-"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"abstract":"<jats:title>Abstract<\/jats:title>\n          <jats:sec>\n            <jats:title>Background:<\/jats:title>\n            <jats:p>Regorafenib, a multitargeted tyrosine kinase inhibitor, proved to be active in patients with soft tissue sarcomas (STS).<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Methods:<\/jats:title>\n            <jats:p>We conducted an open-label, non-randomized, single-center phase II study in advanced pretreated STS patients. Patients received regorafenib 160\u200amg daily on days 1 enrule 21 of a 28-day cycle. The primary endpoint was the progression-free survival (PFS) at 8 weeks. Toxicity was registered.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Results:<\/jats:title>\n            <jats:p>Between April 2015 and November 2016, 21 patients were enrolled in the trial. A total of 13 out of 21 evaluable patients (61.9%) were progression-free at 8 weeks. Median PFS was 3.8 months (95% CI: 2.1\u20139.4). Median overall survival was 14.8 months (95% CI: 7.7\u201327.8). In the intention-to-treat population, we reported a PFS of 66.7% at 3 months (95% CI: 40.4\u201383.4) and 16.7% at 12 months (95% CI: 4.1\u201336.5). As per the RECIST criteria, the response rate was 4.7% (1 partial response out of 21 evaluable patients) with a clinical benefit rate of 61.9%; no complete response was observed. Treatment was well tolerated.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Conclusion:<\/jats:title>\n            <jats:p>Regorafenib shows signs of clinical activity in patients with advanced STS.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Clinical Trial Registration:<\/jats:title>\n            <jats:p>ClinicalTrials.gov NCT02307500.<\/jats:p>\n          <\/jats:sec>","DOI":"10.1097\/md.0000000000020719","type":"journal-article","created":{"date-parts":[[2020,6,23]],"date-time":"2020-06-23T18:42:46Z","timestamp":1592937766000},"page":"e20719","source":"Crossref","is-referenced-by-count":12,"title":["Activity of regorafenib in advanced pretreated soft tissue sarcoma"],"prefix":"10.1097","volume":"99","author":[{"given":"Andrea","family":"Marrari","sequence":"first","affiliation":[{"name":"Medical Oncology and Hematology Unit"},{"name":"Department of Biomedical Sciences, Humanitas University, Pieve Emanuele (Milan), Italy."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alexia","family":"Bertuzzi","sequence":"additional","affiliation":[{"name":"Medical Oncology and Hematology Unit"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Silvia","family":"Bozzarelli","sequence":"additional","affiliation":[{"name":"Medical Oncology and Hematology Unit"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nicol\u00f2","family":"Gennaro","sequence":"additional","affiliation":[{"name":"Department of Radiology"},{"name":"Department of Biomedical Sciences, Humanitas University, Pieve Emanuele (Milan), Italy."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Laura","family":"Giordano","sequence":"additional","affiliation":[{"name":"Biostatistics Unit, Humanitas Cancer Center"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vittorio","family":"Quagliuolo","sequence":"additional","affiliation":[{"name":"Department of Surgery, Humanitas Clinical and Research Center, IRCCS, Rozzano (Milan), Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rita","family":"De Sanctis","sequence":"additional","affiliation":[{"name":"Medical Oncology and Hematology Unit"},{"name":"Department of Biomedical Sciences, Humanitas University, Pieve Emanuele (Milan), Italy."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Simona","family":"Sala","sequence":"additional","affiliation":[{"name":"Medical Oncology and Hematology Unit"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Luca","family":"Balzarini","sequence":"additional","affiliation":[{"name":"Department of Radiology"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1709-9492","authenticated-orcid":false,"given":"Armando","family":"Santoro","sequence":"additional","affiliation":[{"name":"Medical Oncology and Hematology Unit"},{"name":"Department of Biomedical Sciences, Humanitas University, Pieve Emanuele (Milan), Italy."}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"276","published-online":{"date-parts":[[2020,6,26]]},"reference":[{"key":"R1-20230915","doi-asserted-by":"crossref","first-page":"iv51","DOI":"10.1093\/annonc\/mdy096","article-title":"Soft tissue and visceral sarcomas: ESMO-EURACAN Clinical Practice Guidelines for diagnosis, treatment and follow-up","volume":"29","author":"Casali","year":"2018","journal-title":"Ann Oncol"},{"key":"R2-20230915","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1016\/S1470-2045(14)70063-4","article-title":"Doxorubicin alone versus intensified doxorubicin plus ifosfamide for first-line treatment of advanced or metastatic soft-tissue sarcoma: a randomised controlled phase 3 trial","volume":"15","author":"Judson","year":"2014","journal-title":"Lancet Oncol"},{"key":"R3-20230915","doi-asserted-by":"crossref","first-page":"1397","DOI":"10.1016\/S1470-2045(17)30622-8","article-title":"Gemcitabine and docetaxel versus doxorubicin as first-line treatment in previously untreated advanced unresectable or metastatic soft-tissue sarcomas (GeDDiS): a randomised controlled phase 3 trial","volume":"18","author":"Seddon","year":"2017","journal-title":"Lancet Oncol"},{"key":"R4-20230915","doi-asserted-by":"crossref","first-page":"812","DOI":"10.1016\/S1470-2045(17)30334-0","article-title":"Histotype-tailored neoadjuvant chemotherapy versus standard chemotherapy in patients with high-risk soft-tissue sarcomas (ISG-STS 1001): an international, open-label, randomised, controlled, phase 3, multicentre trial","volume":"18","author":"Gronchi","year":"2017","journal-title":"Lancet Oncol"},{"key":"R5-20230915","doi-asserted-by":"crossref","first-page":"786","DOI":"10.1200\/JCO.2015.62.4734","article-title":"Efficacy and safety of trabectedin or dacarbazine for metastatic liposarcoma or leiomyosarcoma after failure of conventional chemotherapy: results of a phase III randomized multicenter clinical trial","volume":"34","author":"Demetri","year":"2016","journal-title":"J Clin Oncol"},{"key":"R6-20230915","doi-asserted-by":"crossref","first-page":"1629","DOI":"10.1016\/S0140-6736(15)01283-0","article-title":"Eribulin versus dacarbazine in previously treated patients with advanced liposarcoma or leiomyosarcoma: a randomised, open-label, multicentre, phase 3 trial","volume":"387","author":"Sch\u00f6ffski","year":"2016","journal-title":"Lancet"},{"key":"R7-20230915","doi-asserted-by":"crossref","first-page":"3154","DOI":"10.1200\/JCO.2008.20.9890","article-title":"Multicenter phase II trial of sunitinib in the treatment of nongastrointestinal stromal tumor sarcomas","volume":"27","author":"George","year":"2009","journal-title":"J Clin Oncol"},{"key":"R8-20230915","doi-asserted-by":"crossref","first-page":"1093","DOI":"10.1093\/annonc\/mds607","article-title":"Phase II prospective study with sorafenib in advanced soft tissue sarcomas after anthracycline-based therapy","volume":"24","author":"Santoro","year":"2013","journal-title":"Ann Oncol"},{"key":"R9-20230915","doi-asserted-by":"crossref","first-page":"3133","DOI":"10.1200\/JCO.2008.20.4495","article-title":"Phase II study of sorafenib in patients with metastatic or recurrent sarcomas","volume":"27","author":"Maki","year":"2009","journal-title":"J Clin Oncol"},{"key":"R10-20230915","doi-asserted-by":"crossref","first-page":"1879","DOI":"10.1016\/S0140-6736(12)60651-5","article-title":"Pazopanib for metastatic soft-tissue sarcoma (PALETTE): a randomised, double-blind, placebo-controlled phase 3 trial","volume":"379","author":"van der Graaf","year":"2012","journal-title":"Lancet"},{"key":"R11-20230915","doi-asserted-by":"crossref","first-page":"303","DOI":"10.1016\/S0140-6736(12)61900-X","article-title":"Regorafenib monotherapy for previously treated metastatic colorectal cancer (CORRECT): an international, multicentre, randomised, placebo-controlled, phase 3 trial","volume":"381","author":"Grothey","year":"2013","journal-title":"Lancet"},{"key":"R12-20230915","doi-asserted-by":"crossref","first-page":"295","DOI":"10.1016\/S0140-6736(12)61857-1","article-title":"Efficacy and safety of regorafenib for advanced gastrointestinal stromal tumours after failure of imatinib and sunitinib (GRID): an international, multicentre, randomised, placebo-controlled, phase 3 trial","volume":"381","author":"Demetri","year":"2013","journal-title":"Lancet"},{"key":"R13-20230915","doi-asserted-by":"crossref","first-page":"56","DOI":"10.1016\/S0140-6736(16)32453-9","article-title":"Regorafenib for patients with hepatocellular carcinoma who progressed on sorafenib treatment (RESORCE): a randomised, double-blind, placebo-controlled, phase 3 trial","volume":"389","author":"Bruix","year":"2017","journal-title":"Lancet"},{"key":"R14-20230915","doi-asserted-by":"crossref","first-page":"1732","DOI":"10.1016\/S1470-2045(16)30507-1","article-title":"Safety and efficacy of regorafenib in patients with advanced soft tissue sarcoma (REGOSARC): a randomised, double-blind, placebo-controlled, phase 2 trial","volume":"17","author":"Mir","year":"2016","journal-title":"Lancet Oncol"},{"key":"R16-20230915","doi-asserted-by":"crossref","first-page":"1753","DOI":"10.1200\/JCO.2006.07.3049","article-title":"Correlation of computed tomography and positron emission tomography in patients with metastatic gastrointestinal stromal tumor treated at a single institution with imatinib mesylate: proposal of new computed tomography response criteria","volume":"25","author":"Choi","year":"2007","journal-title":"J Clin Oncol"},{"key":"R17-20230915","doi-asserted-by":"crossref","first-page":"543","DOI":"10.1016\/S0959-8049(01)00398-7","article-title":"EORTC soft tissue and bone sarcoma group. Progression-free rate as the principal end-point for phase II trials in soft-tissue sarcomas","volume":"38","author":"Van Glabbeke","year":"2002","journal-title":"Eur J Cancer"},{"key":"R18-20230915","doi-asserted-by":"crossref","first-page":"134","DOI":"10.1016\/S1470-2045(18)30676-4","article-title":"Pazopanib for treatment of advanced malignant and dedifferentiated solitary fibrous tumour: a multicentre, single-arm, phase 2 trial","volume":"20","author":"Martin-Broto","year":"2019","journal-title":"Lancet Oncol"}],"container-title":["Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.lww.com\/10.1097\/MD.0000000000020719","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,16]],"date-time":"2023-09-16T00:23:15Z","timestamp":1694823795000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.lww.com\/10.1097\/MD.0000000000020719"}},"subtitle":["Results of a single-center phase II study"],"short-title":[],"issued":{"date-parts":[[2020,6,26]]},"references-count":17,"journal-issue":{"issue":"26","published-online":{"date-parts":[[2020]]}},"URL":"https:\/\/doi.org\/10.1097\/md.0000000000020719","relation":{},"ISSN":["0025-7974","1536-5964"],"issn-type":[{"type":"print","value":"0025-7974"},{"type":"electronic","value":"1536-5964"}],"subject":[],"published":{"date-parts":[[2020,6,26]]}}}