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新辅助 BRAF 靶向治疗局部晚期和寡转移黑色素瘤。

Neoadjuvant BRAF-targeted therapy in regionally advanced and oligometastatic melanoma.

机构信息

Department of Cutaneous Oncology, Moffitt Cancer Center, Tampa, Florida.

Department of Biostatistics and Bioinformatics, Moffitt Cancer Center, Tampa, Florida.

出版信息

Pigment Cell Melanoma Res. 2020 Jan;33(1):86-95. doi: 10.1111/pcmr.12813. Epub 2019 Sep 12.

Abstract

Current management of locoregional and oligometastatic melanoma is typically with surgery; however, some patients are unable to undergo resection due to location/size of their tumors and/or the anticipated morbidity of the surgery. While there are currently no established guidelines for neoadjuvant therapy in melanoma, neoadjuvant BRAF-targeted therapy may make resection more feasible. A retrospective analysis was conducted of 23 patients with BRAFV600-mutant, stage III/IV melanoma treated with BRAF-targeted therapy prior to surgery, with no adjuvant treatment. Surgical specimens, preoperative imaging, and clinical outcomes were evaluated. Results: Ten of 23 patients (44%) attained a pathologic complete response (pCR), with no correlation between RECIST response based on preoperative imaging and pathologic response. After a median of 43-month follow-up, only 1 patient (10%) with a pCR recurred, while 8 of 13 (62%) patients without a pCR recurred. Patients with a pCR had significantly improved relapse-free (RFS) and overall survival (OS) compared to patients with residual tumor. Neoadjuvant BRAF-targeted therapy is associated with a high pCR rate in patients with stage III-IV melanoma, which may correlate with improved RFS and OS.

摘要

目前,局部区域性和寡转移性黑色素瘤的治疗方法通常是手术;然而,由于肿瘤的位置/大小和/或手术的预期发病率,有些患者无法进行切除。虽然目前黑色素瘤的新辅助治疗尚无既定指南,但新辅助 BRAF 靶向治疗可能使切除更可行。对 23 例接受 BRAF 靶向治疗(术前)的 BRAFV600 突变、III/IV 期黑色素瘤患者进行了回顾性分析,未接受辅助治疗。评估了手术标本、术前影像学和临床结果。结果:23 例患者中有 10 例(44%)达到了病理完全缓解(pCR),术前影像学上的 RECIST 反应与病理反应之间没有相关性。中位随访 43 个月后,仅 1 例(10%)pCR 患者复发,而 13 例(62%)未达 pCR 的患者中有 8 例复发。与残留肿瘤患者相比,pCR 患者的无复发生存(RFS)和总生存(OS)显著改善。新辅助 BRAF 靶向治疗与 III/IV 期黑色素瘤患者的高 pCR 率相关,这可能与 RFS 和 OS 的改善相关。

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Neoadjuvant therapy in melanoma: the next step?黑色素瘤的新辅助治疗:下一步何去何从?
Lancet Oncol. 2018 Feb;19(2):151-153. doi: 10.1016/S1470-2045(18)30016-0. Epub 2018 Jan 18.

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