PMID- 38492275 OWN - NLM STAT- Publisher LR - 20240325 IS - 2059-7029 (Electronic) IS - 2059-7029 (Linking) VI - 9 IP - 4 DP - 2024 Mar 15 TI - REGOMA-OSS: a large, Italian, multicenter, prospective, observational study evaluating the efficacy and safety of regorafenib in patients with recurrent glioblastoma. PG - 102943 LID - S2059-7029(24)00711-7 [pii] LID - 10.1016/j.esmoop.2024.102943 [doi] LID - 102943 AB - BACKGROUND: In the randomized phase II REGOMA trial, regorafenib showed promising activity in patients with recurrent glioblastoma. We conducted a large, multicenter, prospective, observational study to confirm the REGOMA data in a real-world setting. PATIENTS AND METHODS: The major inclusion criteria were histologically confirmed diagnosis of glioblastoma according to the World Health Organization (WHO) 2016 classification and relapse after radiotherapy with concurrent/adjuvant temozolomide treatment, good performance status [Eastern Cooperative Oncology Group performance status (ECOG PS 0-1)] and good liver function. Regorafenib was administered at the standard dose of 160 mg/day for 3 weeks on/1 week off. Brain magnetic resonance imaging was carried out within 14 days before starting regorafenib and every 8-12 weeks. The primary endpoint was overall survival (OS). The secondary endpoints were progression-free survival (PFS), objective response rate, disease control rate (DCR), safety and health-related quality of life. The Response Assessment in Neuro-Oncology (RANO) criteria were used for response evaluation and Common Terminology Criteria for Adverse Events (CTCAE) version 5 for assessment of adverse events (AEs). RESULTS: From September 2020 to October 2022, 190 patients with recurrent glioblastoma were enrolled from 30 cancer centers in Italy: their median age was 58.5 years [interquartile range (IQR) 53-67 years], 68% were male and 85 (44.7%) were in optimal clinical condition (ECOG PS 0). The number of patients taking steroids at baseline was 113 (60%); the second surgery was carried out in 39 (20.5%). O(6)-methylguanine-DNA methyltransferase (MGMT) was methylated in 80 patients (50.3%) and 147 (92.4%) of the patients analyzed had isocitrate dehydrogenase (IDH) wild type. The median follow-up period was 20 months (IQR 15.6-25.5 months). The median OS was 7.9 months ([95% confidence interval (CI) 6.5-9.2 months] and the median PFS was 2.6 months (95% CI 2.3-2.9 months). Radiological response was partial response and stable disease in 13 (7.3%) and 26 (14.6%) patients, respectively, with a DCR of 21.9%. The median number of regorafenib cycles per patient was 3 (IQR 2.0-4.0). Grade 3-4 drug-related adverse events were reported in 22.6% of patients. A dose reduction due to AEs was required in 36% of patients. No deaths were considered as treatment-related AEs. CONCLUSIONS: This large, real-world observational study showed similar OS with better tolerability of regorafenib in patients with relapsed glioblastoma compared with the REGOMA study. CI - Copyright (c) 2024 The Author(s). Published by Elsevier Ltd.. All rights reserved. FAU - Caccese, M AU - Caccese M AD - Department of Oncology, Oncology 1, Veneto Institute of Oncology IOV-IRCCS, Padua. Electronic address: mario.caccese@iov.veneto.it. FAU - Desideri, I AU - Desideri I AD - Department of Experimental and Clinical Biomedical Sciences, Radiation Oncology Unit, University of Florence, Azienda Ospedaliero-Universitaria Careggi, Florence. FAU - Villani, V AU - Villani V AD - Neuro-Oncology Unit, IRCCS Regina Elena National Cancer Institute, Rome. FAU - Simonelli, M AU - Simonelli M AD - Department of Biomedical Sciences, Humanitas University, Milan; Humanitas Clinical and Research Center-IRCCS, Humanitas Cancer Center, Milan. FAU - Buglione, M AU - Buglione M AD - Radiation Oncology Unit, ASST Spedali Civili of Brescia, Brescia. FAU - Chiesa, S AU - Chiesa S AD - Dipartimento di Diagnostica per Immagini, Radioterapia Oncologica ed Ematologia, U.O.C. Radioterapia Oncologica, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome. FAU - Franceschi, E AU - Franceschi E AD - Nervous System Medical Oncology Department, IRCCS Istituto Delle Scienze Neurologiche di Bologna, Bologna. FAU - Gaviani, P AU - Gaviani P AD - Neuro-Oncological Unit, Istituto Neurologico Carlo Besta, Milan. FAU - Stasi, I AU - Stasi I AD - Division of Medical Oncology, Civil Hospital, Livorno. FAU - Caserta, C AU - Caserta C AD - Medical Oncology Department, Santa Maria Hospital, Terni. FAU - Brugnara, S AU - Brugnara S AD - Department of Medical Oncology, Santa Chiara Hospital, Trento. FAU - Lolli, I AU - Lolli I AD - Oncology Unit of National Institute of Gastroenterology 'S. De Bellis', Research Hospital, Castellana Grotte, Bari. FAU - Bennicelli, E AU - Bennicelli E AD - Ospedale Policlinico San Martino, Oncologia Medica 2, Genoa. FAU - Bini, P AU - Bini P AD - Neuroncology Unit, IRCCS 'C. Mondino Foundation', University of Pavia, Pavia. FAU - Cuccu, A S AU - Cuccu AS AD - Medical Oncology, Sassari Hospital, Sassari. FAU - Scoccianti, S AU - Scoccianti S AD - Radioterapia Oncologica, Ospedale Santa Maria Annunziata, Bagno a Ripoli, Florence. FAU - Padovan, M AU - Padovan M AD - Department of Oncology, Oncology 1, Veneto Institute of Oncology IOV-IRCCS, Padua. FAU - Gori, S AU - Gori S AD - Oncology Department, IRCCS Sacro Cuore Don Calabria Hospital, Negrar di Valpolicella. FAU - Bonetti, A AU - Bonetti A AD - Department of Oncology, Mater Salutis Hospital, Legnago. FAU - Giordano, P AU - Giordano P AD - Oncology Unit, Ospedale del Mare, Naples. FAU - Pellerino, A AU - Pellerino A AD - Division of Neuro-Oncology, Department of Neuroscience, City of Health and Science and University of Turin, Turin. FAU - Gregucci, F AU - Gregucci F AD - Department of Radiation Oncology, Miulli General Regional Hospital, Acquaviva delle Fonti. FAU - Riva, N AU - Riva N AD - IRCCS Istituto Romagnolo per lo Studio dei Tumori (IRST) 'Dino Amadori', Meldola. FAU - Cinieri, S AU - Cinieri S AD - Oncology Unit, Ospedale Perrino, Brindisi. FAU - Interno, V AU - Interno V AD - Division of Oncology, Department of Biomedical Sciences and Human Oncology, University of Bari, Bari. FAU - Santoni, M AU - Santoni M AD - Oncology Unit, Macerata Hospital, Macerata. FAU - Pernice, G AU - Pernice G AD - Oncology Unit, Fondazione Istituto G. Giglio, Cefalu. FAU - Dealis, C AU - Dealis C AD - Health Directorate, Azienda Sanitaria dell'Alto Adige, Bolzano. FAU - Stievano, L AU - Stievano L AD - Department of Oncology, Ospedale Civile, Rovigo. FAU - Paiar, F AU - Paiar F AD - Department of Radiation Oncology, Azienda Ospedaliero Universitaria Pisana, University of Pisa, Pisa. FAU - Magni, G AU - Magni G AD - Clinical Research Unit, Veneto Institute of Oncology IOV-IRCCS, Padua, Italy. FAU - De Salvo, G L AU - De Salvo GL AD - Clinical Research Unit, Veneto Institute of Oncology IOV-IRCCS, Padua, Italy. FAU - Zagonel, V AU - Zagonel V AD - Department of Oncology, Oncology 1, Veneto Institute of Oncology IOV-IRCCS, Padua. FAU - Lombardi, G AU - Lombardi G AD - Department of Oncology, Oncology 1, Veneto Institute of Oncology IOV-IRCCS, Padua. LA - eng PT - Journal Article DEP - 20240315 PL - England TA - ESMO Open JT - ESMO open JID - 101690685 SB - IM PMC - PMC10959650 OTO - NOTNLM OT - REGOMA OT - glioblastoma OT - real-world OT - recurrent OT - regorafenib EDAT- 2024/03/17 00:42 MHDA- 2024/03/17 00:42 PMCR- 2024/03/15 CRDT- 2024/03/16 19:02 PHST- 2024/01/11 00:00 [received] PHST- 2024/02/02 00:00 [revised] PHST- 2024/02/16 00:00 [accepted] PHST- 2024/03/17 00:42 [medline] PHST- 2024/03/17 00:42 [pubmed] PHST- 2024/03/16 19:02 [entrez] PHST- 2024/03/15 00:00 [pmc-release] AID - S2059-7029(24)00711-7 [pii] AID - 102943 [pii] AID - 10.1016/j.esmoop.2024.102943 [doi] PST - aheadofprint SO - ESMO Open. 2024 Mar 15;9(4):102943. doi: 10.1016/j.esmoop.2024.102943.