Division of Hematology/Oncology, Department of Medicine, Miller School of Medicine, University of Miami, Miami, FL, 33136, USA.
Sylvester Comprehensive Cancer Center, Division of Hematology/Oncology, Department of Medicine, Miller School of Medicine, University of Miami, Miami, FL, 33136, USA.
Future Oncol. 2017 Oct;13(24):2183-2193. doi: 10.2217/fon-2017-0245. Epub 2017 Oct 6.
Gastrointestinal stromal tumors (GISTs) are the most common GI tract mesenchymal tumors. GIST patients are optimally managed by a precision medicine approach. Herein, we discuss the latest advances in precision medicine and ongoing clinical trials relevant to GIST. Circulating tumor DNA for detection of mutational changes could replace tissue biopsies and radiographic imaging once validated. Most GISTs are KIT/PDGFRα mutated, and despite the good clinical response to imatinib, treatment is generally not curative, more often due to secondary mutations. New mechanisms to bypass this resistance by inhibiting KIT downstream pathways and by targeting multiple KIT or PDGFRα mutations are being investigated. Immunotherapy for GIST patients is in its infancy. These approaches may lead to more effective, less toxic therapies.
胃肠道间质瘤（GISTs）是最常见的胃肠道间叶性肿瘤。通过精准医疗方法可对 GIST 患者进行最佳管理。本文讨论了精准医疗的最新进展和与 GIST 相关的正在进行的临床试验。一旦得到验证，循环肿瘤 DNA 检测突变就可能取代组织活检和影像学检查。大多数 GIST 存在 KIT/PDGFRα 突变，尽管对伊马替尼有良好的临床反应，但治疗通常无法治愈，这更多是由于继发突变所致。目前正在研究通过抑制 KIT 下游途径和针对多个 KIT 或 PDGFRα 突变来绕过这种耐药性的新机制。GIST 患者的免疫疗法仍处于起步阶段。这些方法可能会带来更有效、毒性更小的治疗方法。
