Oral smoothened inhibitor for advanced basal cell carcinoma of the hand: a case report

Smoothened inhibitors have recently emerged as a nonsurgical, targeted therapy for treating advanced basal cell carcinomas (BCC) [8]. This novel small-molecule drug class suppresses Hedgehog pathway signaling by binding the membrane protein, Smoothened [7, 9]. Dysregulation of Hedgehog signaling is a hallmark of BCCs and is thought to play an integral role in maintaining cancer growth [1]. Treatment of locally advanced and metastatic basal cell carcinomas with the Smoothened inhibitor vismodegib demonstrated a 40–50 % response rate in Phase I and II trials, paving the way for FDA approval in early 2012 [10]. This therapy is most useful in patients for whom surgical resection is difficult or contraindicated due to tumor size, location, recurrence after surgery, or co-morbid medical conditions. Here, we report a case of a 43-year-old man with numerous and large BCCs of the hand that led to significant disability. The extent of disease suggested that surgical treatment might result in unacceptable functional results. There was involvement of a significant portion of the surface of the hand, and surgical excision would have created a large soft tissue defect requiring skin grafting, with the potential for scar formation, contractures with limitation of motion, and poor aesthetic outcome. Management with oral vismodegib resulted in dramatic tumor regression, with good functional and aesthetic results.