Smoothened receptor inhibitor vismodegib for the treatment of basal cell carcinoma: a retrospective analysis of efficacy and side effects

Abstract

Background: Smoothened receptor inhibitor vismodegib is indicated to treat locally advanced basal cell carcinoma (laBCC) and lesions in nevoid basal cell carcinoma syndrome (NBCCS). Methods: We treated 11 patientsincluding four NBCCS and seven laBCC patientswith vismodegib at our department. Results: Complete remission was achieved in three cases, without relapse after discontinuation. Two of the aforementioned patients had NBCCS, in their cases further treatment might be needed. Two patients showed improvement, but later passed away due to unrelated conditions. Two patients with laBCC initially showed remission, then the treatment was suspended due to side effects. After re-administration of the drug, loss of efficacy was observed. We did not observe therapy resistance in our NBCCS group. The rest of the patients showed good response to therapy, but have not reached full remission yet. The main side effects of vismodegib were muscle cramps, dysgeusia, nausea and alopecia. The frequency of adverse events did not show significant differences between the patient groups. Conclusions: Our results show that vismodegib therapy is effective in the treatment of BCC; however, side effects are often severe. Since the suspension of treatment can lead to therapy resistance, the management of side effects is of great importance.

Retrospective Real-Life Data, Efficacy and Safety of Vismodegib Treatment in Patients with Advanced and Multiple Basal Cell Carcinoma: 3-Year Experience from a Spanish Center

“…Equivalent efficacy in terms of response rates was found in other real-life clinical studies [10,21,22]. In another prospective observational study with a representative sample of United States patients with laBCC (RegiSONIC), the clinical response rate after treatment with vismodegib was 85.1%, with a median duration of response of 17.5 months [23].…”

Molecular profiling of basal cell carcinomas in young patients

“…An inhibition of the expression of PTCH1 or an aberrant activation of SMO are known to be factors leading to the development of BCC. As a matter of fact, driver variants in SMO and PTCH1 are considered to be predictive biomarkers for the response of BCC patients to the treatment with Vismodegib [ 27 ] (Hh pathway inhibitor). Our study confirms the importance of the molecular diagnosis of BCC patients in guiding their treatment.…”

Common benign and malignant tumours of the aging skin: Characteristics and treatment options

“…Basal cell carcinoma has the highest prevalence among skin cancers and is also the most common human malignancy in the Caucasian race [7]. It is essentially non-metastatic, although its locally advanced form (laBCC) can lead to the severe destruction of surrounding tissue, and even to the death of the patient [29][30][31][32]. The clinical presentation is generally an erythematous, occasionally ulcerated, bleeding plaque or bump with a pearly shine [29] (Fig.…”

“…The basic principle is that if the BCC can be removed surgically or otherwise, this is the recommended option even where there is a chance of causing a moderate aesthetic defect. Although BCC lesions generally grow slowly, they can become enormous and laBCCs can develop, which represent a therapeutic challenge [29][30][31]. In several cases, surgical excision is not feasible or would result in severe functional and aesthetic issues.…”

“…In such cases, the targeted small-molecule SMO inhibitors vismodegib or sonidegib may be the only choice of treatment. If the general condition of an elderly patient permits, this may be a good therapeutic option with significant, but in most cases manageable, side effects [30,31]. Nevertheless,…”