Greta Catani, Stefano Kim, Federico Waisberg, Diego Enrico, Romina Luca, Federico Esteso, Luisina Bruno, Andrés Rodríguez, Marcos Bortz, Berenice Freile, Matías Chacón, Ana Isabel Oviedo Albor, Guillermo Méndez, Ezequiel Slutsky, María Cristina Baiud, Romina Llanos, Ayelen Solonyezny, Luis Basbus, Gerardo Arroyo, Julieta Grasselli, Rosario Pasquinelli, Luciana Bella Quero, María Victoria Faura, Ana Cecilia Adur, Mariano Dioca, Mercedes Tamburelli, Javier Castillo, Juan Manuel O'Connor
{"title":"Patients with Colorectal Cancer and <i>BRAF<sup>V600E</sup></i>-Mutation in Argentina: A Real-World Study-The EMOGI-CRC01 Study.","authors":"Greta Catani, Stefano Kim, Federico Waisberg, Diego Enrico, Romina Luca, Federico Esteso, Luisina Bruno, Andrés Rodríguez, Marcos Bortz, Berenice Freile, Matías Chacón, Ana Isabel Oviedo Albor, Guillermo Méndez, Ezequiel Slutsky, María Cristina Baiud, Romina Llanos, Ayelen Solonyezny, Luis Basbus, Gerardo Arroyo, Julieta Grasselli, Rosario Pasquinelli, Luciana Bella Quero, María Victoria Faura, Ana Cecilia Adur, Mariano Dioca, Mercedes Tamburelli, Javier Castillo, Juan Manuel O'Connor","doi":"10.3390/cancers17061007","DOIUrl":null,"url":null,"abstract":"<p><strong>Background/objectives: </strong>The <i>BRAF</i>-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.</p><p><strong>Methods: </strong>We retrospectively analyze patients from ten oncology centers in Argentina, diagnosed with <i>BRAF<sup>V600E</sup></i>-mutated advanced CRC between January 2014 and July 2023.</p><p><strong>Results: </strong>A total of 161 patients with metastatic CRC and <i>BRAF<sup>V600E</sup></i>-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%.</p><p><strong>Conclusions: </strong>This study represents, to the best of our knowledge, the largest published real-world cohort of <i>BRAF<sup>V600E</sup></i>-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.</p>","PeriodicalId":9681,"journal":{"name":"Cancers","volume":"17 6","pages":""},"PeriodicalIF":4.5000,"publicationDate":"2025-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11941482/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cancers","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.3390/cancers17061007","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"ONCOLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Background/objectives: The BRAF-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 with BRAFV600E-mutated advanced CRC between January 2014 and July 2023.
Results: A total of 161 patients with metastatic CRC and BRAFV600E-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 of BRAFV600E-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突变是结直肠癌(CRC)预后不良的因素。中低收入国家需要关于临床特征、结果和治疗策略的真实数据。本研究旨在描述无进展生存期(PFS),以及一线和二线治疗方案的设置和顺序。方法:回顾性分析2014年1月至2023年7月期间阿根廷10个肿瘤中心诊断为brafv600e突变晚期CRC的患者。结果:共有161例转移性结直肠癌患者伴有brafv600e突变。中位年龄为58.5岁(IQR 47-69), 21.7%为mmr缺陷(dMMR)。其中93.8%的患者接受了一线治疗。中位随访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)。49%的患者在一线最常见的方案是双重化疗加抗vegf。26%的患者在二线治疗中接受了BRAF抑制剂,中位PFS为5.2个月(95% CI 4.9-NR);总有效率(ORR)为10.5%。结论:据我们所知,这项研究代表了拉丁美洲已发表的最大的brafv600e突变CRC现实世界队列。治疗方法的异质性反映了我国在获得高成本药物方面存在障碍。需要合作努力来了解这一亚群患者的特殊特征。
期刊介绍:
Cancers (ISSN 2072-6694) is an international, peer-reviewed open access journal on oncology. It publishes reviews, regular research papers and short communications. Our aim is to encourage scientists to publish their experimental and theoretical results in as much detail as possible. There is no restriction on the length of the papers. The full experimental details must be provided so that the results can be reproduced.