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Journal of the American Academy of Dermatology
Sous presse. Epreuves corrigées par l'auteur. Disponible en ligne depuis le mercredi 29 janvier 2020
Doi : 10.1016/j.jaad.2019.12.002
accepted : 3 December 2019
Efficacy, rate of tumor response, and safety of a short course (12-24 weeks) of oral vismodegib in various histologic subtypes (infiltrative, nodular, and superficial) of high-risk or locally advanced basal cell carcinoma, in an open-label, prospective case series clinical trial

Scott W. Fosko, MD a, b, , Melinda B. Chu, MD, MBA c, Eric Armbrecht, PhD d, Tim Galperin, DO e, Geoffrey A. Potts, MD f, Adam Mattox, DO g, Anastasia Kurta, DO a, Kristen Polito, BS a, Jordan B. Slutsky, MD h, Nicole M. Burkemper, MD a, i, M. Yadira Hurley, MD a, i
a Department of Dermatology, Saint Louis University School of Medicine, St. Louis, Missouri 
b Department of Dermatology, Mayo Clinic Florida, Jacksonville, Florida 
c doctor doctor, LLC, Los Angeles, California 
d Saint Louis University Center for Health Outcomes Research, St. Louis, Missouri 
e Kansas City Veterans Affair Medical Center, Kansas City, Missouri 
f Department of Dermatology, Wayne State University, Dearborn, Michigan 
g Department of Dermatology, University of Minnesota, Minneapolis, Minnesota 
h Division of Mohs Surgery and Cutaneous Oncology, Department of Dermatology, Stony Brook University Hospital, Stony Brook, New York 
i Department of Pathology, Saint Louis University School of Medicine, St. Louis, Missouri 

Correspondence to: Scott W. Fosko, MD, 121 Oceanforest Drive North, Atlantic Beach, FL 32233.121 Oceanforest Drive NorthAtlantic BeachFL32233

Vismodegib demonstrated 60% response rates in the ERIVANCE trial. Basal cell carcinoma has various histopathologies. Their effect on response is unclear.


The purpose of this study was to determine whether basal cell carcinoma histopathology affected vismodegib response.


This phase 2b, single-center, prospective case series study compared the efficacy of vismodegib in infiltrative, nodular, and superficial basal cell carcinomas treated for 12 or 24 weeks in 27 patients. Patients had 1 target lesion and up to 3 nontarget lesions.


Twenty-seven patients were enrolled, with 65 tumors (27 target lesions/38 nontarget lesions). At 24 weeks, most basal cell carcinomas achieved histologic clearance, with positive biopsy results in 10.5% of target lesions, 30.4% of nontarget lesions, and 21.4% overall. No statistical differences were observed between histopathologic subtypes. One hundred percent of patients experienced an adverse event, 94% grade 1 or 2. The most common adverse events were dysgeusia/loss of taste (86%), muscle spasms (82%), and alopecia (71%). Clinically progressive disease during treatment was low (1.5%). Two patients had recurrence within 1 year of treatment.


Limitations included sample size of basal cell carcinoma histopathologic subtypes, sampling punch biopsies, and short follow-up.


Basal cell histopathologic subtype did not significantly affect response to vismodegib. Each subtype was observed to completely respond at 12 weeks of therapy, 24 weeks, or both.

The full text of this article is available in PDF format.

Key words : advanced basal cell carcinoma, adverse events, alopecia, basal cell carcinoma, basal cell carcinoma histopathology, cancer, dysgeusia, Hedgehog pathway inhibitor, high risk basal cell carcinoma, histologic clearance, histologic features, histopathologic subtype of basal cell carcinoma, histopathology, infiltrative, locally advanced basal cell carcinoma, muscle spasms, nodular, safety, short course therapy, short-term therapy, superficial, tolerability, tumor response, vismodegib

 Funding sources: Funding was provided to Saint Louis University by Genentech/Roche, who provided minimal input on study design. Funding from Saint Louis University was provided to the Mayo Clinic.
 Conflicts of interest: During the trial at Saint Louis University, Dr Fosko was on the speaker's bureau and advisory boards for Genentech and participated as the principal investigator in a separate vismodegib clinical trial (MIKIE), with funding provided by Genentech/Roche to Saint Louis University. Drs Chu, Armbrecht, Galperin, Potts, Mattox, Kurta, Slutsky, and Burkemper and Ms Polito have no conflicts of interest to declare.
 This clinical trial was approved by the IRBs of Saint Louis University and Mayo Clinic.
 Reprints not available from the authors.

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