Juhasz Margit L W, Marmur Ellen S
J Drugs Dermatol. 2014 Jun;13(6):729-33.
Vismodegib is a first-in-class, hedgehog-signal inhibitor that is FDA-approved for use with advanced basal cell carcinomas (BCCs) that cannot be removed by either surgical resection or treated with radiation. Release of the drug was fast-tracked because of need for this type of drug, and its overall efficacy in clinical trial by producing either regression or even resolution of advanced BCCs. Compared to placebo, patients using vismodegib have arrested BCC progression, reduced size of BCC, and decreased recurrence of BCC. Unfortunately, vismodegib has notable adverse effects (especially those of alopecia, gastrointestinal, muscle spasms, and dysguesia) that make dermatologists reluctant to prescribe the drug and patients unwilling to undergo therapy. In this article, we tackle this dilemma by comparing the toxicity profile of vismodegib to the adverse effect profiles of other dermatologic chemotherapeutics, immunomodulators, retinoids, and biologics. Considering that many of these drugs carry their own risks and those drugs used to treat advanced melanoma have similar toxicity profiles to that of vismodegib, we hope dermatologists and patients alike will be more willing to try vismodegib as a treatment option for advanced BCCs in the future.
维莫德吉是一种一流的刺猬信号抑制剂，已获美国食品药品监督管理局（FDA）批准，用于治疗无法通过手术切除或放射治疗的晚期基底细胞癌（BCC）。由于对这类药物的需求以及其在临床试验中通过使晚期基底细胞癌消退甚至痊愈所展现出的总体疗效，该药物的审批进程得以加快。与安慰剂相比，使用维莫德吉的患者基底细胞癌进展得到遏制，肿瘤大小缩小，复发率降低。不幸的是，维莫德吉有显著的不良反应（尤其是脱发、胃肠道反应、肌肉痉挛和味觉障碍），这使得皮肤科医生不愿开此药，患者也不愿接受治疗。在本文中，我们通过比较维莫德吉的毒性特征与其他皮肤科化疗药物、免疫调节剂、维甲酸类药物和生物制剂的不良反应特征来解决这一困境。鉴于这些药物中的许多都有自身风险，且用于治疗晚期黑色素瘤的药物与维莫德吉有相似的毒性特征，我们希望皮肤科医生和患者未来都更愿意尝试将维莫德吉作为晚期基底细胞癌的一种治疗选择。
