Orlova Kristina V, Ledin Evgeniy V, Zhukova Natalia V, Orlova Rashida V, Karabina Elena V, Volkonskiy Mikhail V, Stroyakovskiy Daniil L, Yurchenkov Aleksandr N, Protsenko Svetlana A, Novik Alexey V, Vorotilina Ludmila V, Moiseenko Fedor V, Chang Victor L, Kazmin Aleksandr I, Tkachenko Svetlana A, Gamaunov Sergey V, Naskhletashvili David R, Samoylenko Igor V, Vikhrova Anastasia S, Utyashev Igor A, Kharkevich Galina Yu, Petenko Natalia N, Shubina Irina Zh, Demidov Lev V
FSBI "N.N. Blokhin National Medical Research Center of Oncology" of the Ministry of Health of the Russian Federation, 115478 Moscow, Russia.
Association Professional Melanoma Society (MELANOMA.PRO), 119192 Moscow, Russia.
Cancers (Basel). 2021 May 21;13(11):2529. doi: 10.3390/cancers13112529.
Clinical trials of targeted therapy (TT) and immunotherapy (IT) for highly aggressive advanced melanoma have shown marked improvements in response and survival rates. However, real-world data on treatment patterns and clinical outcomes for patients with advanced BRAF V600 mutant melanoma are ultimately scarce. The study was designed as an observational retrospective chart review study, which included 382 patients with advanced BRAF V600 mutant melanoma, who received TT in a real-world setting and were not involved in clinical trials. The data were collected from twelve medical centers in Russia. The objective response rates (ORRs) to combined BRAFi plus MEKi and to BRAFi mono-therapy were 57.4% and 39.8%, respectively. The median progression-free survival (PFS) and median overall survival (OS) were 9.2 months and 22.6 months, respectively, for the combined first-line therapy; 9.4 months and 16.1 months, respectively, for the combined second-line therapy; and 7.4 months and 17.1 months, respectively, for the combined third- or higher-line therapy. Analysis of treatment patterns demonstrated the effectiveness of the combined TT with BRAF plus MEK inhibitors in patients with brain metastases, rare types of BRAF mutations, and across lines of therapy, as well as a well-tolerated and manageable safety profile.
针对高度侵袭性晚期黑色素瘤的靶向治疗（TT）和免疫治疗（IT）的临床试验已显示出应答率和生存率有显著提高。然而，关于晚期BRAF V600突变型黑色素瘤患者治疗模式和临床结局的真实世界数据最终却很匮乏。该研究设计为一项观察性回顾性图表审查研究，纳入了382例晚期BRAF V600突变型黑色素瘤患者，这些患者在真实世界环境中接受了TT治疗且未参与临床试验。数据从俄罗斯的12个医疗中心收集。联合使用BRAFi加MEKi和BRAFi单药治疗的客观缓解率（ORR）分别为57.4%和39.8%。一线联合治疗的中位无进展生存期（PFS）和中位总生存期（OS）分别为9.2个月和22.6个月；二线联合治疗分别为9.4个月和16.1个月；三线或更高线联合治疗分别为7.4个月和17.1个月。对治疗模式的分析表明，BRAF加MEK抑制剂联合TT对脑转移患者、罕见类型的BRAF突变患者以及各线治疗均有效，且安全性良好，耐受性和可控性强。
