Hedgehog Antagonist GDC-0449 Is Effective in the Treatment of Advanced Basal Cell Carcinoma
Hedgehog Antagonist GDC-0449 Is Effective in the Treatment of Advanced Basal Cell Carcinoma
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DOI：
10.1002/lary.21145
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发表时间：
2010-12-01
期刊：
影响因子：
2.6
通讯作者：
Hybarger, C. Patrick
中科院分区：
文献类型：
--
作者：
Amin, Shivan H.;Tibes, Raoul;Hybarger, C. Patrick
目的/假设：为了证明刺猬通路抑制剂GDC-0449在治疗晚期基底细胞癌的疗效。设计研究：Case series.Methods：三名患者在转诊中心治疗局部晚期基底细胞癌，转移，被称为GDC-0449 I期临床试验中的治疗。结果：2例患者的临床和影像学表现完全缓解，1例患者的肿瘤负荷明显减轻，影像学表现为缓慢进展的局部病变。副作用是味觉变化，轻度至中度脱发，肌肉痉挛在patient.Conclusions：GDC-0449显示出显着的抑制活性在晚期基底细胞癌的治疗。
Objectives/Hypothesis: To demonstrate the efficacy of the hedgehog pathway inhibitor GDC-0449 in the treatment of advanced basal cell carcinoma.Design Study: Case series.Methods: Three patients treated in a referral center for locally advanced basal cell carcinoma, one with metastases, were referred for treatment in a GDC-0449 phase I clinical trial. The treatment was once per day continuous therapy with oral GDC-0449.Results: Two patients showed complete clinical and radiologic resolution of disease, whereas one patient had significant reduction in tumor burden with radiologic evidence of slowly progressive local disease. Side effects were taste changes, mild to moderate hair loss, and muscle cramps in one patient.Conclusions: GDC-0449 showed significant inhibitory activity in the treatment of advanced basal cell carcinoma.