Vismodegib improves quality of life in patients with periocular locally advanced basal cell carcinoma: subgroup analysis, STEVIE trial

Objectives To evaluate the effect of locally advanced periocular basal cell carcinoma (POLA-BCC) on health-related quality of life (HRQoL) and the benefit of vismodegib treatment among participants in the Safety Events in Vismodegib (STEVIE) trial between 2011 and 2017. Methods The STEVIE trial was conducted in patients with BCC (all anatomic locations) who were treated with vismodegib in 28-day cycles. Patients completed the Skindex-16, a validated questionnaire for the analysis symptoms, emotions, and functioning, at baseline, on day 1 of cycle 2, on day 1 of cycle 7, and at the end-of-study visit. For the present study, data mining techniques were used to construct an ophthalmic database of the STEVIE study. Skindex-16 scores were compared among patients with POLA-BCC between baseline and follow-up and between patients with POLA-BCC and patients with locally advanced BCC on other sites of the head and face (controls). Results The cohort included 169 patients with POLA-BCC and 428 patients with non-periocular head BCC. Patients with POLA-BCC had a significantly worse overall functioning score at baseline than controls ( p = 0.038) and a lower score specifically in activities of daily living ( p = 0.001). At the last follow-up, patients with POLA-BCC showed significant improvement in scores for functioning (100%), symptoms (100%), and emotions (75%) relative to baseline. Conclusions Secondary analysis of the results of the STEVIE trial showed that the HRQoL of patients with POLA-BCC is significantly impaired and can be greatly improved with vismodegib treatment.