Verma Saurav, Reddy Rohit, Chandrashekhara Sheragaru Hanumanthappa, Shamim Shamim Ahmed, Tripathy Sarthak, Rastogi Sameer
Department of Medical Oncology, Institute Rotary Cancer Hospital, All India Institute of Medical Sciences, New Delhi 110029, India.
Department of Radiodiagnosis, Institute Rotary Cancer Hospital, All India Institute of Medical Sciences, New Delhi 110029, India.
Future Sci OA. 2021 Jan 19;7(4):FSO676. doi: 10.2144/fsoa-2020-0178.
The therapeutic landscape in advanced gastrointestinal stromal tumor has evolved. Avapritinib and ripretinib have now been approved by the US FDA for platelet-derived growth factor alpha D842V-mutant and refractory gastrointestinal stromal tumor patients, respectively. Here we report five patients who have been on avapritinib under an expanded access program. Response assessment was available for four patients - a partial response in two patients and stable disease in one, while one patient had progressive disease. Though preliminary results of the VOYAGER trial have shown less activity of avapritinib and no significant difference in progression-free survival when compared with regorafenib, avapritinib may show some clinical benefit in a subset of patients refractory to approved therapies. We share our experience of five cases, with clinical benefit in three. We believe avapritinib should be further evaluated in clinical trials.
晚期胃肠道间质瘤的治疗格局已经发生了变化。阿伐替尼和瑞派替尼现已分别被美国食品药品监督管理局（FDA）批准用于血小板衍生生长因子α D842V突变型和难治性胃肠道间质瘤患者。在此，我们报告5例在扩大准入项目下接受阿伐替尼治疗的患者。4例患者可进行疗效评估——2例部分缓解，1例疾病稳定，1例疾病进展。尽管VOYAGER试验的初步结果显示，与瑞戈非尼相比，阿伐替尼的活性较低且无进展生存期无显著差异，但阿伐替尼可能对一部分对已批准治疗方案难治的患者显示出一定的临床益处。我们分享5例患者的经验，其中3例有临床获益。我们认为阿伐替尼应在临床试验中进一步评估。
