Pirozzi Mario, Caterino Marianna, Facchini Sergio, Zotta Alessia, Messina Gaetana, Rauso Raffaele, Sica Antonello, Sciano Donato, Facchini Gaetano, Orditura Michele, Somma Teresa, Maiuri Francesco, Cappabianca Paolo, Ciardiello Fortunato, Fasano Morena
Department of Precision Medicine, University of Campania "Luigi Vanvitelli,", Naples, Italy.
Thoracic Surgery Unit, Università degli Studi della Campania "Luigi Vanvitelli,", Naples, Italy.
Case Rep Oncol. 2022 Jun 27;15(2):642-647. doi: 10.1159/000524954. eCollection 2022 May-Aug.
Glioblastoma multiforme (GBM) is one of the most frequent and aggressive primary tumors in the central nervous system, representing more than 60% of all brain tumors in adults. Primary GBM remains incurable with a poor prognosis both for limited therapeutic alternatives and for a high risk of progression or recurrence. In fact, at recurrence, the few treatment options available, and often characterized by limited effectiveness, have always been an Achilles' heel. The recent approval of second line of regorafenib, a multikinase inhibitor, has given hope after several years of darkness for new therapies in the treatment of GBM. Indeed, in the REGOMA trial, a phase 2 study, regorafenib was the first drug to show a statistically significant improvement in median overall survival compared with lomustine group, usually used in the second-line treatment after temozolomide failure. We report a case of a 43-year-old patient affected by GBM in treatment with regorafenib in third line of therapy with good disease control and long PFS.
多形性胶质母细胞瘤（GBM）是中枢神经系统中最常见且侵袭性最强的原发性肿瘤之一，占成人所有脑肿瘤的60%以上。原发性GBM仍然无法治愈，预后较差，这是因为治疗选择有限，且进展或复发风险高。事实上，在复发时，可用的治疗选择很少，而且往往效果有限，这一直是一个致命弱点。多激酶抑制剂瑞戈非尼二线治疗方案最近获得批准，在多年的黑暗之后，为GBM治疗的新疗法带来了希望。的确，在2期研究REGOMA试验中，与常用于替莫唑胺治疗失败后二线治疗的洛莫司汀组相比，瑞戈非尼是首个显示中位总生存期有统计学显著改善的药物。我们报告了一例43岁的GBM患者，其在三线治疗中使用瑞戈非尼，疾病得到良好控制，无进展生存期长。
