Department of Pathology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark.
Department of Radiology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark.
Lung Cancer. 2017 Nov;113:14-17. doi: 10.1016/j.lungcan.2017.08.024. Epub 2017 Sep 1.
Patients with epidermal growth factor receptor (EGFR) gene-mutated non-small cell lung cancer (NSCLC) obtain substantial clinical benefit from EGFR tyrosine-kinase inhibitors (TKIs), but will ultimately develop TKI-resistance resulting in median progression-free survival of 9-15 months during first-line TKI-therapy. However, type and timing of TKI-resistance cannot be predicted and several mechanisms may simultaneously/subsequently occur during TKI-treatment. In this respect, we present a 49 year-old Caucasian male ex-smoker with metastatic pulmonary adenocarcinoma (ADC) that concomitantly harbored an EGFR exon 19-mutation (p.E746_A750delELREA) and a previously unreported 2bp frame-shift microdeletion in the fibroblast growth factor receptor 3 (FGFR3; p.D785fs*31) gene. Interestingly, FGFR3-mutations have previously been described in other cancer types of Caucasian patients and may represent an alternative pathway to EGFR-signaling. The patient received first-line erlotinib but after only 7 weeks showed metastatic pleural effusion, in which transformation to small cell lung cancer (SCLC) that retained the EGFR- and FGFR3-mutations was identified. Consequently, standard carboplatin-etoposide regimen for SCLC combined with erlotinib continuation was implemented obtaining significant objective response. However, after completing 6 cycles of this combination, new pulmonary and hepatic metastases appeared and showed persistence of the original EGFR- and FGFR3-mutated ADC phenotype together with acquisition of the erlotinib-resistant T790M EGFR-mutation. The patient rapidly deteriorated and deceased. Thus, this advanced EGFR-mutated NSCLC displayed very rapid onset and heterogeneous genetic and phenotypic mechanisms of TKI-resistance occurring at different times and locations of metastatic disease: concomitant FGFR3-mutation before and during TKI-treatment as potential intrinsic mechanism for the rapid progression; transformation to SCLC at first progression during TKI-therapy; acquired T790M EGFR-mutation at second progression. Our case also underlines that, when achievable, rebiopsies of progressive sites during TKI-treatment are important for identifying heterogeneous histopathological and molecular resistance mechanisms and better defining possible treatment modifications.
表皮生长因子受体 (EGFR) 基因突变的非小细胞肺癌 (NSCLC) 患者从 EGFR 酪氨酸激酶抑制剂 (TKI) 中获得显著的临床获益,但最终会产生 TKI 耐药性,导致一线 TKI 治疗期间无进展生存期中位数为 9-15 个月。然而,TKI 耐药的类型和时间无法预测,并且在 TKI 治疗期间可能同时或随后发生几种机制。在这方面,我们介绍了一位 49 岁的白种男性曾吸烟者,患有转移性肺腺癌 (ADC),同时携带 EGFR 外显子 19 突变 (p.E746_A750delELREA) 和先前未报道的成纤维细胞生长因子受体 3 (FGFR3) 基因中的 2bp 框移微缺失 (p.D785fs*31)。有趣的是,FGFR3 突变已在其他白种人群的癌症类型中描述过,可能代表 EGFR 信号的另一种途径。该患者接受了一线厄洛替尼治疗,但仅 7 周后就出现了转移性胸腔积液,其中转化为保留 EGFR 和 FGFR3 突变的小细胞肺癌 (SCLC)。因此,实施了标准的卡铂依托泊苷方案联合厄洛替尼继续治疗,获得了显著的客观缓解。然而,在完成该联合治疗 6 个周期后,新出现了肺部和肝脏转移,并显示出原始 EGFR 和 FGFR3 突变的 ADC 表型的持续存在,同时获得了对厄洛替尼耐药的 T790M EGFR 突变。患者病情迅速恶化并死亡。因此,这种晚期 EGFR 突变的 NSCLC 表现出非常快速的发生和 TKI 耐药的异质性遗传和表型机制,这些机制发生在转移疾病的不同时间和部位:在 TKI 治疗前和治疗期间同时发生 FGFR3 突变作为快速进展的潜在内在机制;在 TKI 治疗期间首次进展时转化为 SCLC;在第二次进展时获得 T790M EGFR 突变。我们的病例还强调,当可行时,在 TKI 治疗期间对进展部位进行再活检对于识别异质的组织病理学和分子耐药机制以及更好地确定可能的治疗修改非常重要。