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 language | English (US) |
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Pages (from-to) | 946-954 |
Number of pages | 9 |
Journal | Journal of the American Academy of Dermatology |
Volume | 82 |
Issue number | 4 |
DOIs | |
State | Published - 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