同时携带表皮生长因子受体（EGFR）突变和并发突变的非小细胞肺癌（NSCLC）患者预后较差。本研究旨在研究阿罗替尼联合埃可替尼作为EGFR突变的NSCLC携带者的一线治疗选择，无论是否存在并发突变。本研究为2期单臂、多中心试验（ClinicalTrials.gov NCT03736837），于2018年12月至2020年11月在中国的五家医院进行。以EGFR敏感突变的非鳞状细胞非小细胞肺癌病例接受了阿罗替尼和埃可替尼治疗。主要终点为无进展生存期（PFS）。次要终点包括客观缓解率（ORR）、疾病控制率（DCR）、总生存期（OS）和毒性。共招募了60名参与者，其中31名（52%）患有并发突变，29名（48%）患有有病理意义的并发突变。中位随访时间为26.9个月（范围：15.0-38.9个月）。ORR为68.5%，DCR为98.2%。中位PFS为15.1个月（95%CI: 12.6-17.6个月），符合主要终点；中位DoR为13.5个月（95%CI: 10.0-17.1个月），中位OS为30.0个月（95%CI: 25.5-34.5个月）。携带有病理意义并发突变的患者的中位PFS和OS分别为15.6个月（95%CI: 12.5-18.7个月）和未达到中位OS（95%CI: 17.46个月至未达到），所有患者均出现治疗相关不良事件（TRAEs），其中26名（43%）和1名（1.7%）患者出现≥3级和严重的治疗相关不良事件（TRAEs）。阿罗替尼和埃可替尼联合治疗作为EGFR突变阳性的晚期NSCLC的一线治疗选择疗效显著，耐受性良好。ClinicalTrials.gov识别号：NCT03736837。© 2023. BioMed Central Ltd., Springer Nature的一部分。

Non-small cell lung cancer (NSCLC) patients with epidermal growth factor receptor (EGFR) mutation and concurrent mutations have a poor prognosis. This study aimed to examine anlotinib plus icotinib as a first-line treatment option for advanced NSCLC carrying EGFR mutation with or without concurrent mutations.This phase 2, single-arm, multicenter trial (ClinicalTrials.gov NCT03736837) was performed at five hospitals in China from December 2018 to November 2020. Non-squamous NSCLC cases with EGFR-sensitizing mutations were treated with anlotinib and icotinib. The primary endpoint was progression-free survival (PFS). Secondary endpoints included the objective response rate (ORR), disease control rate (DCR), overall survival (OS), and toxicity.Sixty participants were enrolled, including 31 (52%) and 29 (48%) with concurrent mutations and pathogenic concurrent mutations, respectively. The median follow-up was 26.9 (range, 15.0-38.9) months. ORR and DCR were 68.5% and 98.2%, respectively. Median PFS was 15.1 (95%CI: 12.6-17.6) months which met the primary endpoint, median DoR was 13.5 (95%CI: 10.0-17.1) months, and median OS was 30.0 (95%CI: 25.5-34.5) months. Median PFS and OS in patients with pathogenic concurrent mutations were 15.6 (95%CI: 12.5-18.7) months and not reached (95%CI: 17.46 months to not reached), respectively. All patients experienced TRAEs, including 26 (43%) and 1 (1.7%) who had grade ≥ 3 and serious treatment-related adverse events (TRAEs).Anlotinib combined with icotinib was effective and well-tolerated as a first-line treatment option for EGFR mutation-positive advanced NSCLC with or without concurrent mutations.ClinicalTrials.gov identifier: NCT03736837.© 2023. BioMed Central Ltd., part of Springer Nature.