Department of Medical Oncology, Leiden University Medical Center, 2300 RC, Leiden, The Netherlands.
Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden, The Netherlands.
Target Oncol. 2022 Nov;17(6):627-634. doi: 10.1007/s11523-022-00926-6. Epub 2022 Nov 14.
The prognosis of patients with advanced gastrointestinal stromal tumor (GIST) has improved greatly after the introduction of imatinib. However, primary or secondary resistance to imatinib occurs in the majority of patients. Sunitinib is the standard second line treatment in exon-9 mutated GIST.
We compared the clinical outcomes of sunitinib with imatinib dose escalation in patients with progressive advanced non-KIT exon 9 mutated GIST after failure of first line imatinib.
A retrospective study was performed, retrieving data from a real-life database (Dutch GIST Registry) including patients with GIST treated with sunitinib or imatinib dose escalation after failure on first line imatinib 400 mg daily. Primary outcome measures were progression free survival (PFS) and overall survival (OS).
In total, 110 patients were included, 72 (65.5%) patients were treated with sunitinib (group A) and 38 (34.5%) received an imatinib dose escalation (group B). Important prognostic features at baseline, such as tumor size, stage at diagnosis, mitotic count and localization were equally distributed in both groups. No significant difference (p = 0.88) between median PFS in group A [8.7 months (95% CI 5.6-11.3)] and group B [5.6 months, (95% CI 2.6-8.7)] was observed. Moreover, the OS was similar between group A and group B; 63.2 months and 63.4 months, respectively.
This study represents a proper sample size cohort containing detailed data on mutational status of patients with advanced GIST. We illustrated that imatinib dose escalation could serve as a good alternative for sunitinib as second-line treatment in patients with a non-KIT exon 9 mutation.
伊马替尼的引入极大地改善了晚期胃肠道间质瘤（GIST）患者的预后。然而，大多数患者会出现对伊马替尼的原发性或继发性耐药。舒尼替尼是exon-9 突变 GIST 的标准二线治疗药物。
我们比较了伊马替尼剂量递增与舒尼替尼治疗一线伊马替尼治疗失败后进展的晚期非 KIT exon 9 突变 GIST 患者的临床结局。
进行了一项回顾性研究，从真实世界数据库（荷兰 GIST 登记处）中检索了接受舒尼替尼或伊马替尼剂量递增治疗的 GIST 患者的数据，这些患者在一线伊马替尼 400mg 每日治疗失败后。主要终点为无进展生存期（PFS）和总生存期（OS）。
共纳入 110 例患者，72 例（65.5%）患者接受舒尼替尼治疗（A 组），38 例（34.5%）接受伊马替尼剂量递增治疗（B 组）。两组患者的基线重要预后特征，如肿瘤大小、诊断时的分期、有丝分裂计数和定位，分布均匀。A 组中位 PFS [8.7 个月（95%CI 5.6-11.3）]与 B 组 [5.6 个月，（95%CI 2.6-8.7）]之间无显著差异（p = 0.88）。此外，A 组和 B 组的 OS 相似；分别为 63.2 个月和 63.4 个月。
本研究代表了一个合适的样本量队列，包含了晚期 GIST 患者突变状态的详细数据。我们表明，伊马替尼剂量递增可作为非 KIT exon 9 突变患者二线治疗的舒尼替尼的良好替代药物。
