Long-term safety, tolerability, and efficacy of vismodegib in two patients with metastatic basal cell carcinoma and basal cell nevus syndrome.
Long-term safety, tolerability, and efficacy of vismodegib in two patients with metastatic basal cell carcinoma and basal cell nevus syndrome.
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DOI：
10.4081/dr.2011.e55
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发表时间：
2011-10-05
影响因子：
1.1
通讯作者：
Von Hoff DD
中科院分区：
文献类型：
--
作者：
Weiss GJ;Tibes R;Blaydorn L;Jameson G;Downhour M;White E;Caro I;Von Hoff DD
在使用vismodegib（一种SMO拮抗剂）的临床试验中观察到晚期基底细胞癌（BCC）的肿瘤缓解。SMO拮抗作用的结果是抑制Hedgehog Signaling Pathway（HHSP）下游靶基因。HHSP抑制已显示影响负责血液、乳腺和神经发育的干细胞。我们报告我们的经验，治疗两个病人与先进的BCC参与。这两名患者至少2.25年没有新的BCC发展。这两名患者都接受了超过2.75年的vismodegib每日治疗，没有出现任何明显的副作用。在长期连续每日给药SMO拮抗剂后，我们没有观察到血液学参数的显著变化或两名晚期BCC患者胸部的身体异常。
Tumor responses in advanced basal cell carcinoma (BCC) have been observed in clinical trials with vismodegib, a SMO antagonist. The result of SMO antagonism is inhibition Hedgehog Signaling Pathway (HHSP) downstream target genes. HHSP inhibition has been shown to affect stem cells responsible for blood, mammary, and neural development. We report on our experience of treating two patients with advanced BCC participating. These two patients have had no new BCCs develop for at least 2.25 years. Both patients have been receiving ongoing daily treatment with vismodegib for greater than 2.75 years without experiencing any significant side effects. After prolonged continuous daily dosing with a SMO antagonist, we have not observed a significant alteration in hematologic parameters or physical abnormalities of the pectoral regions of two patients with advanced BCC.