Treatment of locally advanced or multiple basal cell carcinoma with vismodegib in real world setting.

Abstract

e22065 Background: Evaluation of efficacy and safety of vismodegib (V) was done in a retrospective analysis of patients (pts) with locally advanced or multiple basal cell carcinoma (laBCC or multiple BCC) and pts with Goltz-Gorlin Syndrom (G-G Syn) in routine clinical practice. Methods: Baseline characteristics, efficacy data and treatment-related adverse events were collected from 30 laBCC or multiple BCC and 6 G-G Syn pts who were treated with V. Results: In 86-month period, 36 pts were diagnosed with laBCC (18 pts), multiple BCC (12 pts) or G-G Syn (6 pts), all inappropriate for surgery or radiotherapy. Baseline characteristics: median age was 72.6 years in laBCC + multiple BCC pts group and 51.3 years in G-G Syn pts group. Sixty percent of pts in laBCC + multiple BCC group were females; majority (70%) of pts were previously treated by surgery (S) and/or radiotherapy (RT); 43% of pts had 1 lesion with predominant localization in central face (eyes, nose, lips or ears in 84% of pts), 20% had 2-3 lesions and 37% more than 3 lesions. Fifty percent of pts in G-G Syn pts group were males; all pts were previously treated with S and/or RT. The overall response rate (ORR) was 76% in laBCC + multiple BCC and 83% in G-G Syn pts group. Disease control rate (DCR) was 93% in laBCC + multiple BCC and 100% in G-G Syn pts group. Median duration of treatment (DoT) was 7.8 months (range: 1.3-29.8) in laBCC + multiple BCC group and 27.1 months (range: 4.8-86.4) in G-G Syn group. At the time of analysis in laBCC or multiple BCC group one patient died due to other reasons than cancer, in 30% of pts treatment has been interrupted during the treatment course [in most cases due to complete response or adverse events (AEs)], 40% of pts are still on treatment. In G-G Syn group treatment has been interrupted in 50% of pts (in most cases due to adverse events), 67% of pts are still on treatment. AEs of any grade were reported in 97% of pts in laBCC or multiple BCC group and 83% in G-G Syn group. Majority of AEs in laBCC or multiple BCC group were grade 1 or 2 (96%)., 4% of AEs were grade 3: muscle cramps in 3 pts, respiratory infection, vomiting and anemia in 1 patient each. Majority of AEs in G-G Syn group were also grade 1 or 2 (87%), 13% of AEs were grade 3: muscle cramps in 2 pts, weight loss and diarrhea in 1 patient each. No grade 4 or 5 AEs were reported. Conclusions: Vismodegib has shown meaningful efficacy with manageable safety profile in pts with laBCC or multiple BCC as well as in pts with G-G Syn in real world setting.