Gastrointestinal stromal tumours (GIST) are the most common mesenchymal tumours of the gastrointestinal tract. Surgical treatment is recommended for the majority of localised GIST, while systemic treatment is the cornerstone of management for metastatic or unresectable disease. While a three-year regimen of imatinib is the standard of care in the adjuvant setting, there is no precise recommendation for the duration of neoadjuvant treatment, where imatinib is usually given between 4 and 12 months. Continuous treatment with imatinib at a dose of 400 mg once per day is recommended for most patients with unresectable or metastatic GIST in the first line. An exception is represented by patients with tumours harbouring the imatinib-insensitivePDGFRAD842V mutation who would be better treated with avapritinib. Targeted therapies are also recommended in the presence of NTRK rearrangements and BRAF mutations, although limited data are available. While an increase in the dose of imatinib to 800 mg is an option for the second line, sunitinib is usually considered the standard of care. Similar outcomes were reported for ripretinib in patients with tumours harbouring KIT exon 11 mutation, with significantly fewer side effects. Regorafenib and ripretinib are the standards of care in the third and fourth lines, respectively. The recent development of various systemic treatment options allows for a more personalised approach based on the molecular profile of the GIST, patient characteristics, and the profile of medications’ adverse events. A multidisciplinary approach is paramount since combining systemic treatment with locoregional treatment options and supportive care is vital for long-term survival.
胃肠道间质瘤（GIST）是胃肠道最常见的间叶源性肿瘤。对于大多数局限性GIST，推荐采用手术治疗；而对于转移性或不可切除的GIST，全身性治疗则是管理的基石。在辅助治疗中，伊马替尼的三年疗程是标准治疗方案，但新辅助治疗的持续时间尚无明确推荐，通常伊马替尼的使用时间为4至12个月。对于一线治疗中大多数不可切除或转移性GIST患者，推荐每日一次400毫克伊马替尼的持续治疗。例外情况是携带伊马替尼不敏感PDGFRAD842V突变的患者，这类患者更适合接受阿伐普替尼治疗。尽管现有数据有限，但在存在NTRK重排和BRAF突变的情况下，也推荐使用靶向治疗。在二线治疗中，将伊马替尼剂量增加至800毫克是一种选择，但舒尼替尼通常被视为标准治疗。对于携带KIT外显子11突变的患者，瑞派替尼显示出相似的疗效，且副作用显著减少。瑞戈非尼和瑞派替尼分别是三线和四线治疗的标准方案。近年来，多种全身治疗方案的进展使得基于GIST的分子特征、患者特点以及药物不良反应谱的个体化治疗成为可能。多学科协作至关重要，因为将全身治疗与局部区域治疗方案及支持性护理相结合，对于患者的长期生存至关重要。
Evaluation of Systemic Treatment Options for Gastrointestinal Stromal Tumours
肿瘤(癌症)患者之家