Medical Oncology Department, Institut Català d'Oncologia, L'Hospitalet de Llobregat, Spain.
Badalona Applied Research Group in Oncology, B-ARGO, Spain.
Mol Oncol. 2023 Sep;17(9):1908-1916. doi: 10.1002/1878-0261.13444. Epub 2023 Jun 12.
Efficiency of expanded genomic profiling (EGP) programmes in terms of final inclusion of patients in genomically matched therapies is still unknown. Fit patients with advanced and refractory colorectal cancer (CRC) were selected for an EGP programme. Next-generation sequencing (NGS) analysis from formalin-fixed paraffin-embedded tumour samples was performed. The purpose was to describe the prevalence of genomic alterations defined by the ESMO Scale for Clinical Actionability of Molecular Targets (ESCAT), as well as the percentage of patients finally included in genomically guided clinical trials. In total, 187 patients were recruited. Mutational profile was obtained in 177 patients (10 patients were failure due to insufficient tumour sample), copy number alterations in 41 patients and fusions in 31 patients. ESCAT-defined alterations were detected in 28.8% of the intention-to-analyse population. BRAF V600E was clustered in ESCAT I, with a prevalence of 3.7%, KRAS G12C and ERBB2 amplification were clustered in ESCAT II, whose prevalence was 4.2% and 1.6%, respectively. Most alterations were classified in ESCAT III (mutations in ERBB2, PIK3CA or FGFR genes and MET amplification) and IV (mutations in BRAF non-V600E, ERBB3, FBXW7, NOTCH, RNF43), with a single prevalence under 5%, except for PIK3CA mutation (9%). The final rate of inclusion into genomically guided clinical trials was 2.7%, including therapies targeting BRAF V600E or RNF43 mutations in two patients each, and ERBB2 mutation in one patient. In conclusion, EGP programmes in patients with advanced CRC are feasible and identify a subset of patients with potentially druggable genomic alterations. However, further efforts must be made to increase the rate of patients treated with genomically guided therapies.
在接受基因组匹配治疗的患者中,扩展基因组分析(EGP)计划的最终纳入效率尚不清楚。选择晚期和难治性结直肠癌(CRC)的合适患者进行 EGP 计划。对福尔马林固定石蜡包埋肿瘤样本进行下一代测序(NGS)分析。目的是描述 ESMO 分子靶向临床可操作性量表(ESCAT)定义的基因组改变的流行率,以及最终纳入基因组指导临床试验的患者比例。共招募了 187 名患者。在 177 名患者中获得了突变谱(由于肿瘤样本不足,10 名患者失败),41 名患者存在拷贝数改变,31 名患者存在融合。在意向分析人群中,检测到 ESCAT 定义的改变占 28.8%。BRAF V600E 聚集在 ESCAT I 中,患病率为 3.7%,KRAS G12C 和 ERBB2 扩增聚集在 ESCAT II 中,患病率分别为 4.2%和 1.6%。大多数改变被归类为 ESCAT III(ERBB2、PIK3CA 或 FGFR 基因中的突变和 MET 扩增)和 IV(非 BRAF V600E、ERBB3、FBXW7、NOTCH、RNF43 中的突变),单一患病率低于 5%,除了 PIK3CA 突变(9%)。最终纳入基因组指导临床试验的比例为 2.7%,包括针对 2 名患者的 BRAF V600E 或 RNF43 突变和 1 名患者的 ERBB2 突变的治疗。总之,晚期 CRC 患者的 EGP 计划是可行的,并确定了一组具有潜在可用药基因组改变的患者。然而,必须进一步努力提高接受基因组指导治疗的患者比例。