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, Melinda B. Chu, Eric Armbrecht, Tim Galperin, Geoffrey A. Potts, Adam Mattox, Anastasia Kurta, Kristen Polito, Jordan B. Slutsky, Nicole M. Burkemper, M. Yadira Hurley

Research output: Contribution to journalArticlepeer-review

11 Scopus citations

Abstract

Background: Vismodegib demonstrated 60% response rates in the ERIVANCE trial. Basal cell carcinoma has various histopathologies. Their effect on response is unclear. Objective: The purpose of this study was to determine whether basal cell carcinoma histopathology affected vismodegib response. Methods: 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. Results: 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: Limitations included sample size of basal cell carcinoma histopathologic subtypes, sampling punch biopsies, and short follow-up. Conclusions: 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.

Original languageEnglish (US)
Pages (from-to)946-954
Number of pages9
JournalJournal of the American Academy of Dermatology
Volume82
Issue number4
DOIs
StatePublished - Apr 2020

Bibliographical note

Funding Information:
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.

Publisher Copyright:
© 2019 American Academy of Dermatology, Inc.

Keywords

  • Hedgehog pathway inhibitor
  • advanced basal cell carcinoma
  • adverse events
  • alopecia
  • basal cell carcinoma
  • basal cell carcinoma histopathology
  • cancer
  • dysgeusia
  • 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

PubMed: MeSH publication types

  • Clinical Trial, Phase II
  • Journal Article

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