A phase II, multicenter, open-label, 3-cohort trial evaluating the efficacy and safety of vismodegib in operable basal cell carcinoma - 17/06/15
Abstract |
Background |
Vismodegib is approved for treatment of advanced basal cell carcinoma.
Objective |
We sought to characterize vismodegib efficacy and safety in operable basal cell carcinoma.
Methods |
Patients with new, operable, nodular basal cell carcinoma received vismodegib (150 mg/d) followed by excision and Mohs micrographic surgery to ensure clear margins. Cohort 1 received vismodegib for 12 weeks; cohort 2 received vismodegib for 12 weeks, then 24 weeks of observation before excision; and cohort 3 received vismodegib for 8 weeks on/4 weeks off/8 weeks on.
Results |
In all, 24 patients enrolled in cohort 1, and 25 in cohorts 2 and 3. Complete histologic clearance was achieved by 42%, 16%, and 44% of patients in cohorts 1, 2, and 3, respectively. Muscle spasms (76%), alopecia (58%), and dysgeusia (50%) were the most frequent adverse events (AEs). Five (7%) patients discontinued treatment because of an AE. AE reversibility was evaluated in cohort 2 with 24 weeks of observation after treatment discontinuation.
Limitations |
Nonrandomized, small cohort sizes, and short observation durations for some patients are limitations.
Conclusion |
Primary efficacy end points were not met (predefined complete histologic clearance rate: >50% in cohorts 1 and 3; >30% in cohort 2). Safety was comparable when dosed continuously versus intermittently. Posttreatment reversibility of vismodegib-related AEs was demonstrated.
Le texte complet de cet article est disponible en PDF.Key words : basal cell carcinoma, hedgehog pathway inhibitor, vismodegib
Abbreviations used : AE, BCC, CHC, FDA, SAE
Plan
Funded by Genentech, a member of the Roche Group. Financial support for editorial assistance was provided by Roche. |
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Disclosure: Dr Sofen is as an advisory board member, consultant, investigator, and speaker for Genentech, receiving honoraria. Drs Gross and Sharata are investigators for Genentech. Dr Hamilton is an investigator for Roche. Dr Egbert is a consultant for Genentech, receiving salary. Dr Lyons and Dr Hou are employees of Genentech, receiving salary, stock, and stock options. Dr Caro is an employee and stock holder of Genentech, receiving salary, stock, and stock options. Dr Goldberg has no conflicts of interest to declare. |
Vol 73 - N° 1
P. 99 - juillet 2015 Retour au numéroBienvenue sur EM-consulte, la référence des professionnels de santé.
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