Nilotinib has been used as a third-line drug for gastrointestinal stromal tumors (GISTs) after a failure of sunitinib. In this study, we aimed to evaluate the efficacy of nilotinib in different genomic subtypes of GISTs. We searched the English articles through EMBASE, Cochrane Library and PubMed Database regarding to the use of nilotinib on GISTs, which published up to February 15, 2019. Inclusion criteria were: GISTs patients received nilotinib in a clinical trial and had detailed genetic subtype records (such as KIT exon 9, KIT exon 11, or PDGFRA mutations, or wild-type). The clinical benefit rate was used to assess the efficacy of nilotinib. A total of 3 studies involving 218 GISTs were included in this meta-analysis. The overall OR (KIT group vs WT group) was 3.26 (95% CI: 1.14-9.28; P = 0.027, P_{heterogeneity} = 0.613). The overall OR in KIT exon 11 group vs WT group was 5.30 (95% CI: 1.79-15.68; P = 0.003, P_{heterogeneity} = 0.409). The overall OR in KIT exon 9 group vs WT group was 0.13 (95% CI: 0.02-0.86; P = 0.035, P_{heterogeneity} = 0.229). The overall OR in KIT exon 11 group vs exon 9 group was 9.96 (95% CI: 0.39-254.66; P < 0.0001, P_{heterogeneity} = 0.024). Different genotypes of GISTs showed different responses to nilotinib, and KIT exon 11-mutant GISTs mostly benefited from nilotinib, followed by KIT exon 9-mutant or WT one.

在舒尼替尼失败后，尼罗替尼已被用作胃肠道间质瘤 (GIST) 的三线药物。在本研究中，我们旨在评估尼罗替尼在不同基因组亚型 GIST 中的疗效。我们通过 EMBASE、Cochrane 图书馆和 PubMed 数据库检索了截至 2019 年 2 月 15 日发表的关于尼罗替尼在 GIST 上的使用的英文文章。纳入标准是：GIST 患者在临床试验中接受了尼罗替尼，并且有详细的遗传亚型记录（例如 KIT 外显子 9、KIT 外显子 11 或 PDGFRA 突变或野生型）。临床受益率用于评估尼罗替尼的疗效。该荟萃分析共纳入 3 项研究，涉及 218 例 GIST。总体 OR（KIT 组与 WT 组）为 3.26（95% CI：1.14-9.28；P  = 0.027，P_异质性 = 0.613）。KIT 外显子 11 组与 WT 组的总体 OR 为 5.30（95% CI：1.79-15.68；P  = 0.003，P_异质性 = 0.409）。KIT 外显子 9 组与 WT 组的总体 OR 为 0.13（95% CI：0.02-0.86；P  = 0.035，P_异质性 = 0.229）。KIT 外显子 11 组与外显子 9 组的总体 OR 为 9.96（95% CI：0.39-254.66；P < 0.0001，P_异质性 = 0.024）。不同基因型的 GIST 对尼罗替尼表现出不同的反应，KIT 外显子 11 突变 GIST 主要受益于尼罗替尼，其次是 KIT 外显子 9 突变或 WT 1。