维莫德吉治疗肿瘤反应的初始率，可以预测眼睑 LA-BCC 的完全缓解结果。
The initial rate of tumour response to vismodegib treatment, can predict a complete response outcome for periocular LA-BCC.
机构信息
Department of Ophthalmology, Rabin Medical Center - Beilinson Hospital, Petach Tikva, Israel.
Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
出版信息
Eye (Lond). 2023 Feb;37(3):531-536. doi: 10.1038/s41433-022-01982-y. Epub 2022 Feb 24.
PURPOSE
To establish a model to predict treatment outcome of periocular locally advanced basal cell carcinoma (POLA BCC) based on initial response to treatment with vismodegib (Erivedge), a sonic hedgehog inhibitor.
DESIGN
Subgroup analysis of data from the STEVIE study database.
METHODS
Analysis of medical history, treatment protocol, and treatment outcome of POLA BCC tumours in a STEVIE study population of 244 POLA BCC patients treated with ≥1 dose of vismodegib.
RESULTS
A predictive model for complete response (CR) was established based on the initial treatment response. A cutoff value of 20% reduction in tumour size at 3 months of treatment identified the patients with a high probability (82.76%) to achieve CR. A second cutoff value of 67.7% reduction in tumour size at 6 months of treatment improved the prediction to a 95.42% probability of a CR outcome.
CONCLUSIONS
A treatment model was constructed based on the prediction of a CR outcome and the initial response to vismodegib treatment at 3 and 6 months. The study result provide significant new insights can facilitate decision-making on treatment management according to tumour response in patients with POLA BCC.
目的
基于 sonic hedgehog 抑制剂维莫德吉（Erivedge）治疗的初始反应，建立预测眶周局部晚期基底细胞癌（POLA BCC）治疗结果的模型。
设计
STEVIE 研究数据库中的数据亚组分析。
方法
对 244 例接受维莫德吉治疗≥1 剂的 POLA BCC 患者的 STEVIE 研究人群的病史、治疗方案和治疗结果进行分析。
结果
根据初始治疗反应建立了完全缓解（CR）的预测模型。治疗 3 个月时肿瘤大小缩小 20%的截断值可识别出有高概率（82.76%）实现 CR 的患者。治疗 6 个月时肿瘤大小缩小 67.7%的第二个截断值可将预测结果提高到 95.42%的 CR 结局概率。
结论
根据治疗 3 个月和 6 个月时 CR 结局的预测和维莫德吉治疗的初始反应，构建了治疗模型。研究结果提供了重要的新见解，可以根据 POLA BCC 患者的肿瘤反应来促进治疗管理决策。
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