title: Vismodegib Therapy for Basal Cell Carcinoma in an 8-Year-Old Chinese Boy with Xeroderma Pigmentosum.
abstract: Vismodegib is an oral inhibitor of the Hedgehog signaling pathway and has been used to treat basal cell carcinoma (BCC) in adults. This article reports clearance of a nodular BCC of the nasal tip in an 8-year-old boy with xeroderma pigmentosum (XP). BCC can pose therapeutic challenges when located in areas that are not amenable to traditional therapies such as Mohs micrographic surgery or topical agents. Vismodegib was used at a dose of 150 mg/day to treat the boy's BCC. After 4 months of therapy, we achieved complete clinical clearance. During 21 months of follow-up, the patient's nose remained clinically clear of tumor. Vismodegib was successfully used to treat a child with XP and nodular BCC. Our goal in using vismodegib was tumor regression while avoiding cosmetic and functional disfigurement. Vismodegib was effective in clinically clearing the tumor, and the patient has shown no signs of recurrence. Further studies are warranted.
authors.affiliation: Surgical Dermatology, Las Vegas, Nevada.
authors.forename: Douglas
authors.initials: D
authors.lastname: Fife
authors.affiliation: School of Medicine, University of Nevada, Reno, Nevada.
authors.forename: Marko A
authors.initials: MA
authors.lastname: Laitinen
authors.affiliation: Nellis Air Force Base, Mike O'Callaghan Federal Medical Center, Las Vegas, Nevada.
authors.forename: David J
authors.initials: DJ
authors.lastname: Myers
authors.affiliation: Nellis Air Force Base, Mike O'Callaghan Federal Medical Center, Las Vegas, Nevada.
authors.forename: Pamela B
authors.initials: PB
authors.lastname: Landsteiner
mesh_terms: D000813:Anilides / Q000627:therapeutic use*; D002280:Basal Cell Carcinoma / Q000188:drug therapy* / Q000473:pathology*; D002648:Child; D005153:Facial Neoplasms / Q000188:drug therapy* / Q000473:pathology; D006801:Humans; D008297:Male; D009666:Nose; D011725:Pyridines / Q000627:therapeutic use*; D012878:Skin Neoplasms / Q000188:drug therapy* / Q000473:pathology; D014983:Xeroderma Pigmentosum / Q000150:complications* / Q000473:pathology
chemical_list: D000813:Anilides; C538724:HhAntag691; D011725:Pyridines