| Title | In Defence of Vismodegib: A Multicentre Case Series |
| Submitted by | Huw Oliphant |
| Abstract Number | 336 |
| 19-218 | |
| Review Result | poster presentation |
| Purpose |
Vismodegib is a first-in-class Hh (Hedgehog) pathway inhibitor, previously available for the treatment of basal cell carcinoma (BCC). It has recently been withdrawn by the National Institute for Health and Care Excellent (NICE) due to apparent lack of benefit, and questions over cost-effectiveness. The purpose of this study is to present a multicentre case series of patients treated for advanced periocular BCC using Vismodegib. |
| Methods |
Six patients were included in this multicentre case series (Brighton, Cardiff, Newcastle). Basic demographic data was obtained using a standard data collection sheet, alongside BCC characteristics, nature of orbital involvement (if any), response to treatment, side effects, and adjuvant treatment, |
| Results |
Average age was 75 (61-90). Four tumours were deep ulcerating lesions at the medial or lateral acanthi, and 2 were irregularly defined tumours. Three patients (50%) demonstrated orbital involvement. Three patients represented recurrent disease (50%). One patient had full regression of the BCC, and 4 had a partial response (1 patient had a lack of data to determine response). Five out of six patients experienced side effects, though most frequently fatigue was encountered. Two patients underwent subsequent resection and one patient radiotherapy. Mean Follow up period was 27 months (11-78 months). |
| Conclusion |
Whilst Vismodegib has been withdrawn from use in the National Health Service, there remains a strong case for its use in selected scenarios. In particular, it is likely to have utility in advanced BCC with orbital invasion, where globe sparing surgery is sought. |
Additional Authors
| Last name | Initials | City / Hospital | Department |
|---|---|---|---|
| Edmunds | Matthew | Cardiff/University Hospital of Wales | Ophthalmology |
| Haridas | A | Cardiff/University Hospital of Wales | Ophthalmology |
| Althaus | M | Newcastle/Royal Victoria Infirmary | Ophthalmology |
| Clarke | L | Newcastle/Royal Victoria Infirmary | Ophthalmology |
| Laybourne | J | Manchester/Manchester Royal Eye Hospital | Ophthalmology |
| Rajak | S | Brighton/Sussex Eye Hospital |