Background/Objectives: TheBRAF-mutation is a poor prognostic factor in colorectal cancer (CRC). There is a need for real-world data in low-middle-income countries regarding clinical characteristics, outcomes, and treatment strategies. This study aims to describe progression-free survival (PFS) and in the first- and second-line setting and sequences of treatment regimens. Methods: We retrospectively analyze patients from ten oncology centers in Argentina, diagnosed withBRAFV600E-mutated advanced CRC between January 2014 and July 2023. Results: A total of 161 patients with metastatic CRC andBRAFV600E-mutation. The median age was 58.5 (IQR 47–69), and 21.7% were MMR-deficient (dMMR). Of these patients, 93.8% received first-line treatment. With a median follow-up of 23 months (95% CI 16.5–33.4 months), the median PFS was 9 months (95% CI 7.4–10.5 months). The most common regimen in first line setting was doublet chemotherapy plus anti-VEGF for 49% of the patients. Twenty-six percent of the patients received BRAF inhibitors in the second-line setting, with a median PFS of 5.2 months (95% CI 4.9—NR); the overall response rate (ORR) was 10.5%. Conclusions: This study represents, to the best of our knowledge, the largest published real-world cohort ofBRAFV600E-mutated CRC in Latin America. The heterogeneity of the treatments reflects the existence of barriers to access to high-cost drugs in our country. Cooperative efforts are needed to understand the particular characteristics of this subgroup of patients.
背景/目的：BRAF基因突变是结直肠癌（CRC）的不良预后因素。中低收入国家亟需关于BRAF突变结直肠癌患者临床特征、治疗结局及治疗策略的真实世界数据。本研究旨在描述一线及二线治疗背景下患者的无进展生存期（PFS）以及治疗方案序列。方法：我们回顾性分析了2014年1月至2023年7月期间阿根廷十家肿瘤中心诊断为BRAF V600E突变晚期结直肠癌的患者。结果：共纳入161例BRAF V600E突变转移性结直肠癌患者。中位年龄为58.5岁（四分位距47-69），其中21.7%为错配修复缺陷（dMMR）。在这些患者中，93.8%接受了一线治疗。中位随访时间为23个月（95% CI 16.5-33.4个月），中位PFS为9个月（95% CI 7.4-10.5个月）。一线治疗中最常见的方案是双药化疗联合抗血管内皮生长因子（VEGF）治疗，占患者的49%。26%的患者在二线治疗中接受了BRAF抑制剂治疗，中位PFS为5.2个月（95% CI 4.9—未达到）；总体缓解率（ORR）为10.5%。结论：据我们所知，本研究代表了拉丁美洲地区已发表的最大规模BRAF V600E突变结直肠癌真实世界队列。治疗方案的异质性反映了我国在获取高成本药物方面存在的障碍。需要协同努力以深入了解该亚组患者的独特特征。
肿瘤(癌症)患者之家