Experience With Vismodegib in the Treatment of Advanced Basal Cell Carcinoma at a Cancer Center

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 were recorded. Overall survival (OS) and progression-free survival (PFS) were evaluated with Kaplan-Meier analysis. The median follow-up period was 17 months (range: 1.6-57.3), and the median age at diagnosis 73 years (range: 39-88). The most common disease location was head and neck (86.2%), and the most common non-skin sites 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. The number of metastatic patients was 5 (17.2%). With vismodegib treatment, the complete response rate was 27.6%, partial response 55.2%, and stable response 10.3%. Treatment responses were most frequently seen within 2 months from the beginning of vismodegib. 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), and 12.1 ± 4.6 months (2.9-21.2) in the mBCC. In the univariable analysis for the OS, only the treatment after the vismodegib was statistically significant, showing chemotherapy was better comparing to no treatment or surgery. The most common adverse events were fatigue-58.6%, muscle spasms-48.3%, alopecia-13.8%, and weight loss-13.8%. This real-life data study shows that vismodegib treatment in locally advanced and metastatic BCC was well tolerated and effective.

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“…8 In a retrospective study involving 22 patients, objective response rates were 86%. 10 In our result, objective response rates…”

Section: Discussionsupporting

confidence: 51%

Efficacy and tolerability of vismodegib treatment in locally advanced and metastatic basal cell carcinoma: Retrospective real‐life data

Gürbüz

Doğan

Akkuş

et al. 2021

Dermatologic Therapy

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“…Therefore, surgery continues to play an important role in achieving long-term remission. Investigations into vismodegib’s potential as a neoadjuvant treatment of aBCC are ongoing, 9,13 including VISMONEO (ClinicalTrials.gov identifier: NCT02667574).…”

Section: Discussionmentioning

confidence: 99%

“…The reported experience with aBCC treated with vismodegib is variable, and there remain unanswered questions regarding the optimal use in the context of general clinical practice, 9 and optimal duration of treatment to balance outcomes with adverse effects has yet to be established. 10…”

Section: Introductionmentioning

confidence: 99%

Real-World Experience of Vismodegib in Advanced Basal Cell Carcinoma at a Canadian Cancer Center

et al. 2021

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Background Vismodegib is a novel Hedgehog pathway inhibitor that has revolutionized the treatment of patients with advanced basal cell carcinoma (BCC) who are poor candidates for surgery or radiation. Few studies have explored the use of vismodegib to facilitate further surgery or radiotherapy, and the optimal treatment duration to balance outcomes with adverse effects. Objectives To characterize the disease response, progression, and recurrence outcomes of BCC patients, and to report the impact of subsequent therapies. Methods We performed a retrospective study of 46 adult patients with advanced basal cell carcinoma (aBCC), including both locally advanced (laBCC) and metastatic (mBCC) disease, treated with vismodegib at a single center from 2012 to 2019. Results Thirty-six had laBCC, and 10 had mBCC. Treatment was given over a mean of 21.9 months. Twenty-three (50%) had a complete response (CR), and 19 (41.3%) achieved partial response (PR). Median time to maximal response was 5.3 months. Eleven (23.9%) had resected disease at median 17.2 months, and 11 patients (23.9%) received radiotherapy. Thirty-two (69.6%) experienced progressive disease after achievement of CR or PR. Among 17 CR patients, who stopped treatment, 14 (82.3%) experienced subsequent relapse; 6 (85%) attained a repeat response. Twenty (43.5%) discontinued treatment at least once due to adverse effects. Conclusions With a response rate of 91%, London Regional Cancer Center’s (LRCP)’s experience with vismodegib supports its effectiveness in treatment of aBCC. Moreover, a significant number of patients treated with vismodegib became amenable to surgery or radiotherapy. Toxicity remained an important factor that limited treatment duration.

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“…Although vismodegib is widely used for the treatment of advanced BCC, further research has been advocated on its effectiveness and treatment schedules in clinical practice (12,13). In particular, some concerns on the use of vismodegib have been raised due to the frequent onset of AEs associated with this molecule, especially cramps and dysgeusia (16)(17)(18).…”

Section: Discussionmentioning

confidence: 99%

“…Vismodegib is an oral inhibitor of hedgehog pathway, approved for locally advanced and metastatic BCC in patients who are not amenable to surgery or radiation (5)(6)(7)(8)(9). Some fieldpractice experiences have evaluated the use of vismodegib for the treatment of BCC in daily practice (10)(11)(12)(13)(14)(15). However, the description of a further series of patients will help towards a more comprehensive evaluation of this treatment.…”

Section: Introductionmentioning

confidence: 99%

Tailored Toxicity-Driven Administration of Vismodegib in Patients With Multiple or Locally Advanced Basal Cell Carcinoma: A Pilot Analysis

et al. 2020

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Experience With Vismodegib in the Treatment of Advanced Basal Cell Carcinoma at a Cancer Center

Cited by 6 publications

References 20 publications

Efficacy and tolerability of vismodegib treatment in locally advanced and metastatic basal cell carcinoma: Retrospective real‐life data

Efficacy and tolerability of vismodegib treatment in locally advanced and metastatic basal cell carcinoma: Retrospective real‐life data

Real-World Experience of Vismodegib in Advanced Basal Cell Carcinoma at a Canadian Cancer Center

Tailored Toxicity-Driven Administration of Vismodegib in Patients With Multiple or Locally Advanced Basal Cell Carcinoma: A Pilot Analysis

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