Vismodegib treatment in locally advanced basal cell carcinoma limited to the facial region: a single-center experience.

IF 3.4 2区 医学 Q2 ONCOLOGY
İvo Gökmen, Erdem Şen
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引用次数: 0

Abstract

Introduction: Basal cell carcinoma (BCC) is the most common non-melanoma skin cancer. Treatment typically begins with surgical intervention. However, in cases of locally advanced and metastatic BCC (laBCC and mBCC), Hedgehog signaling pathway inhibitors such as sonidegib and vismodegib are used.

Materials and methods: This retrospective study included 28 adult patients with laBCC who were treated with 150 mg daily oral vismodegib at Mehmet Akif Ersoy State Hospital between 2018 and 2023. Patients were monitored until disease progression, intolerable side effects, or treatment discontinuation. Treatment efficacy was evaluated using objective response rate (ORR) and disease control rate (DCR). Safety was assessed based on adverse events (AEs) reported in accordance with the CTCAE v. 5.0 classification. Statistical analyses, including overall survival (OS) and progression-free survival (PFS), were performed and analyzed using SPSS version 25.

Results: The overall ORR was 89.3%, with 39.3% of patients achieving complete response (CR). The median PFS was 15.1 months, and the median OS was 37.5 months. Female gender and prior surgical interventions were identified as independent prognostic factors for PFS, while ECOG-PS score and the duration of vismodegib exposure were significant predictors of OS. AEs were reported in 78.6% of patients, with dysgeusia and muscle spasms being the most prevalent. Grade 3 or 4 toxicity occurred in 14.5% of patients, and 13% (n = 3) discontinued treatment due to AEs. Patients receiving treatment for ≥ 12 months experienced a higher incidence of AEs (92.9%) compared to those treated for < 12 months (64.3%).

Conclusion: Vismodegib demonstrated high efficacy in treating patients with laBCC, with a significant proportion achieving CR. However, long-term treatment was associated with an increased incidence of AEs, highlighting the need for careful monitoring of safety in prolonged therapies.

Vismodegib治疗仅限于面部区域的局部晚期基底细胞癌:单中心经验。
基底细胞癌(BCC)是最常见的非黑色素瘤皮肤癌。治疗通常从手术干预开始。然而,在局部晚期和转移性BCC (laBCC和mBCC)的情况下,使用刺猬信号通路抑制剂,如sonidegib和vismodegib。材料和方法:本回顾性研究纳入了2018年至2023年在Mehmet Akif Ersoy州立医院接受每日150mg口服vismodegib治疗的28例laBCC成年患者。对患者进行监测,直至疾病进展、出现无法忍受的副作用或停止治疗。采用客观缓解率(ORR)和疾病控制率(DCR)评价治疗效果。安全性根据CTCAE v. 5.0分级报告的不良事件(ae)进行评估。统计分析,包括总生存期(OS)和无进展生存期(PFS),使用SPSS版本25进行分析。结果:总ORR为89.3%,39.3%的患者达到完全缓解(CR)。中位PFS为15.1个月,中位OS为37.5个月。女性性别和既往手术干预被确定为PFS的独立预后因素,而ECOG-PS评分和vismodegib暴露时间是OS的重要预测因素。78.6%的患者出现不良反应,其中发音困难和肌肉痉挛最为常见。14.5%的患者出现了3级或4级毒性,13% (n = 3)的患者因不良反应而停止治疗。结论:Vismodegib在治疗laBCC患者中表现出很高的疗效,达到CR的比例很高。然而,长期治疗与ae发生率增加相关,强调了在长期治疗中需要仔细监测安全性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
BMC Cancer
BMC Cancer 医学-肿瘤学
CiteScore
6.00
自引率
2.60%
发文量
1204
审稿时长
6.8 months
期刊介绍: BMC Cancer is an open access, peer-reviewed journal that considers articles on all aspects of cancer research, including the pathophysiology, prevention, diagnosis and treatment of cancers. The journal welcomes submissions concerning molecular and cellular biology, genetics, epidemiology, and clinical trials.
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