Efficacy and safety of regorafenib in Japanese patients with advanced gastrointestinal stromal tumors.
Efficacy and safety of regorafenib in Japanese patients with advanced gastrointestinal stromal tumors.
复制标题
瑞戈非尼对日本晚期胃肠道间质瘤患者的疗效和安全性。
DOI：
10.1007/s10147-022-02159-z
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发表时间：
2022
期刊：
影响因子：
--
通讯作者：
Doki Y.
中科院分区：
文献类型：
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作者：
Teranishi R;Takahashi T;Nishida T;Hirota S;Kurokawa Y;Saito T;Yamamoto K;Yamashita K;Tanaka K;Makino T;Motoori M;Omori T;Nakajima K;Eguchi H;Doki Y.
研究背景Regorafenib是一种口服多激酶抑制剂，在伊马替尼和舒尼替尼失效后，已被确定为患者的三线治疗药物。然而，由于日本人群中瑞格非尼的临床数据仍然缺乏，所以瑞格非尼的管理主要是根据每个肿瘤学家的临床经验。方法对33例转移性和复发性胃肠道间质瘤患者应用瑞可非尼治疗，观察其抗肿瘤效果，包括总生存期、无进展生存期和安全性，并根据不良事件的发生率进行评价。结果瑞格拉非尼治疗患者的中位总生存期为23.8个月，1年总生存率为80.0%，中位无进展生存期为7.1个月，1年无进展生存率为40.2%。瑞格非尼部分缓解3例(9.1%)，稳定17例(51.5%)，进展10例(30.3%)，无效3例(9.1%)。疾病控制率为54.0%。所有患者都报告了与治疗相关的不良事件，最常见的是手足综合征(72.7%)，其次是肝损害(36.4%)和腹泻(27.3%)，6例(20.0%)患者因不良事件而停用。瑞格非尼在日本晚期胃肠道间质瘤患者中显示出有效性和可控的安全性，这与以前的研究相当。
BackgroundRegorafenib is an oral multi-kinase inhibitor that has been established as third-line treatment for patients after the failure of imatinib and sunitinib. However, since clinical data of regorafenib in the Japanese population are still lacking, the management of regorafenib is mainly based on the clinical experience of each oncologist. The aim of this study was to evaluate the efficacy and safety of regorafenib in a Japanese population.MethodsThirty-three patients treated with regorafenib for metastatic and recurrent gastrointestinal stromal tumors were retrospectively enrolled. This study investigated the anti-tumor effect, including overall survival, progression-free survival, and safety, which was evaluated based on the incidence of adverse events.ResultsThe median overall survival of patients treated with regorafenib was 23.8 months and the 1-year overall survival rate was 80.0%, the median progression-free survival was 7.1 months and the 1-year progression-free survival rate was 40.2%. The responses to regorafenib were partial response in 3 cases (9.1%), stable disease in 17 (51.5%), progressive disease in 10 (30.3%), and non-evaluable in 3 (9.1%). The disease control rate was 54.0%. Treatment-related adverse events were reported in all patients, with the most common being hand-foot syndrome (72.7%), followed by liver damage (36.4%) and diarrhea (27.3%), and six patients (20.0%) were discontinued due to adverse events.ConclusionThis is the first report of Japanese patients with gastrointestinal stromal tumors treated with regorafenib. Regorafenib showed efficacy and a manageable safety profile in Japanese patients with advanced gastrointestinal stromal tumors, which was comparable with previous studies.