Avapritinib治疗血小板源性生长因子受体α外显子18突变的晚期胃肠间质瘤的研究进展

Research Progress of Avapritinib in the Treatment of Advanced Gastrointestinal Stromal Tumors with Mutation of Exon 18 of Platelet-Derived Growth Factor Receptor α

摘要: 胃肠间质瘤(GIST)是一类起源于胃肠道间叶组织的肿瘤，可根据编码受体酪氨酸激酶蛋白(KIT)和血小板源性生长因子受体α (PDGFRA)突变进行分子分类。酪氨酸激酶抑制剂(tyrosine kinase inhibitor, TKI)是胃肠间质瘤全身治疗的基础药物，显著延长了晚期胃肠间质瘤患者的生存期，但PDGFRA外显子18突变的晚期GIST对传统的靶向药物耐药。而Avapritinib是一种有效的KIT和PDGFRA-特异性酪氨酸激酶抑制剂，对PDGFRA外显子18 D842V突变的胃肠间质瘤患者显示出良好的缓解率，为耐药的晚期胃肠间质瘤患者提供了更多的治疗机会。本文对Avapritinib治疗PDGFRA外显子18 D842V突变的晚期胃肠间质瘤的研究进展进行综述。

Abstract: Gastrointestinal stromal tumors (GIST) are a group of tumors originating from the stromal lobe of the gastrointestinal tract that can be molecularly classified by mutations encoding the receptor ty-rosine kinase protein (KIT) and platelet-derived growth factor receptor α (PDGFRA). Tyrosine ki-nase inhibitors (tyrosine kinase inhibitor, TKI) are the basic drugs for the systemic treatment of gastrointestinal GISTs, significantly prolonging the survival of patients with advanced gastrointes-tinal GISTs, but the advanced GIST with PDGFRA exon 18 mutation is resistant to conventional tar-geted drugs. Avapritinib, a potent KIT and PDGFRA-specific tyrosine kinase inhibitor, showed a fa-vorable response rate for patients with PDGFRA exon 18 D842V mutation, providing more thera-peutic opportunities for resistant patients with advanced gastrointestinal GISTs. This paper reviews Avapritinib treatment for advanced gastrointestinal stromal tumors with PDGFRA exon 18 D842V mutation.