Original articleAn investigator-initiated open-label clinical trial of vismodegib as a neoadjuvant to surgery for high-risk basal cell carcinoma
Section snippets
Methods
All patients meeting study inclusion criteria were offered participation (N = 17). We enrolled 15 patients over 18 years of age with at least 1 biopsy-confirmed BCC of any histologic subtype, more than 5 mm in diameter, eligible for surgical removal, and who agreed to participate in an investigator-initiated, open-label, single-arm intervention trial (NCT01631331) from April 2012 to July 2013 at a single institution. Patients with previously treated/recurrent BCCs were also eligible. We
Patients
We enrolled 15 patients; 11 patients completed the trial and had their target BCCs surgically excised (10 by Mohs and 1 by standard excision). One patient was lost to follow-up, 2 patients withdrew from the trial because of vismodegib-related side effects (elevated creatine phosphokinase, fatigue), and 1 patient withdrew because of unrelated adverse events (ventriculoperitoneal shunt obstruction) (Fig 1 [CONSORT diagram]). The mean age of the 11 patients was 59 (range 39-100) years (Table I).
Discussion
Neoadjuvant treatment with vismodegib for an average of 4 months before surgery reduced tumor area and surgical defect size.10 This reduction was seen primarily in nonrecurrent tumors. Efficacy appears to be duration dependent as a minimum of 3 months of vismodegib was required. Hair loss, muscle cramps, and dysgeusia cannot be avoided even with short-term use. Clinical appearance of tumors after vismodegib was variable and did not predict histologic cure.
To date, there have been no clinical
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Dr Ally and Dr Aasi contributed equally to this work.
This investigator-initiated trial was supported in part by a Damon Runyon Cancer Research Foundation Clinical Investigator Award (CI-54-11 to Dr Tang).
Disclosure: Drs Tang, Chang, and Oro have been investigators in studies sponsored by Genentech. Dr Oro has also been an investigator in studies sponsored by Infinity and Novartis and Dr Chang is also a clinical investigator for studies sponsored by Novartis and Lilly. Drs Ally, Aasi, Wysong, and Kim; Ms Teng; Mr Anderson; and Ms Bailey-Healy have no conflicts of interest to declare.