Basal cell carcinoma (BCC) is the most common cancer. Surgical excision is the primary treatment of BCC. Radiotherapy and topical therapies are other treatment options. Vismodegib inhibits the hedgehog signaling pathway that is an important mechanism of developing BCC. The study aims to evaluate the vismodegib treatment in local advanced (laBCC) and metastatic (mBCC) basal cell carcinoma.
The data of 29 patients were retrospectively reviewed. The clinical and histopathological features of the patients and adverse events of vismodegib treatment were recorded. Overall survival (OS) and progression-free survival (PFS) evaluated with Kaplan-Meier analysis.
The median follow-up period was 17 months (range: 1.6-57.3). The median age at diagnosis was 73 years (range: 39-88). The percentages of female and male patients were 51.7% and 48.3%, respectively. The most common location of disease was head and neck (86.2%). The number of metastatic patients was 5 (17.2%). The most common non-skin locations of disease were lymph nodes-13.8%, bone-13.8%, lung-6.9%, and brain-6.9%. Three (10.3%) patients had Gorlin’s syndrome. Before vismodegib treatment, 15 (51.7%) patients had received radiotherapy and 18 (62.1%) patients had undergone surgery. With vismodegib treatment, the complete response rate was 27.6% and partial response 55.2%, and stable response 10.3%. Treatment responses were most frequently seen within two months from the beginning of vismodegib. The most common adverse events were fatigue-58.6%, muscle spasms-48.3%, alopecia-13.8%, and weight loss- 13.8%. A total of 8 (27.6%) patients deceased during the study period. The median OS was 43.3±9.0 months (25.6-61.1) for all patients. The median PFS in the laBCC was 15.7±1.8 months (12.2-19.3). The median PFS in the mBCC was 12.1±4.6 months (2.9-21.2). After the disease progression under vismodegib treatment, 53.8% of the patients received chemotherapy or had surgical treatment.
In this study, we determined that vismodegib treatment in locally advanced and metastatic BCC was well tolerated and effective.
The authors.
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All authors have declared no conflicts of interest.