Patient With Gorlin Syndrome and Metastatic Basal Cell Carcinoma Refractory to Smoothened Inhibitors

Zhu, Gefei Alex; Li, Angela S.; Chang, Anne Lynn S.

doi:10.1001/jamadermatol.2013.8744

Cited by 14 publications

(13 citation statements)

References 13 publications

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“…The cost of vismodegib (approximately US $7500 per month) is another limiting factor of compliance. Sonedigib, which has a similar mechanism of action and side effects as vismodegib, can also be used …”

Section: Treatmentmentioning

confidence: 99%

“…Sonedigib, which has a similar mechanism of action and side effects as vismodegib, can also be used. 37,61,62 Itraconazole, a systemic antifungal, is an SMO inhibitor that has potential for use in BCNS. Specifically, itraconazole prevents SMO from migrating and accumulating at the tip of the cilia after hedgehog signalling stimulation.…”

Section: Targeting Geneticsmentioning

confidence: 99%

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Basal cell naevus syndrome: an update on genetics and treatment

John

Schwartz

2015

Br J Dermatol

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Basal cell naevus syndrome is an autosomal dominant disorder that stems from mutations in multiple genes, most commonly patched 1 (PTCH1). The classic triad of symptoms consists of basal cell carcinomas, jaw keratocysts and cerebral calcifications, although there are many other systemic manifestations. Because of the broad range of symptoms and development of several types of tumours, early diagnosis and close monitoring are essential to preserve quality of life. Targeting treatment is often difficult because of tumour prevalence. Newer inhibitors of the hedgehog signalling pathway and proteins involved in proliferative growth have shown therapeutic promise. In addition, preventive medications are being devised. We propose a method for determining appropriate treatment for cutaneous tumours.

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Section: Treatmentmentioning

confidence: 99%

Section: Targeting Geneticsmentioning

confidence: 99%

Basal cell naevus syndrome: an update on genetics and treatment

John

Schwartz

2015

Br J Dermatol

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“…Given the expected need for prolonged vismodegib treatment in patients with multiple recurrent BCCs, researchers have raised concerns of acquired resistance, as recently described in patients with Gorlin syndrome. 5 In conclusion, vismodegib offers an efficient and relatively safe therapeutic alternative for recurrent radiation-induced locally advanced BCCs that are not amenable to surgery or radiotherapy. …”

Section: Discussionmentioning

confidence: 96%

Vismodegib for radiation-induced multiple basal cell carcinomas (BCCs) of the scalp

Tauber

Pavlovsky

Fenig

et al. 2015

Journal of the American Academy of Dermatology

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“…The progression of the disease should be distinguished from primary resistance in which the tumor never responds to treatment. Both primary and secondary resistance has been reported in sporadic and NBCC patients (30)(31)(32). Resistance for vismodegib is hypothesized to account for mutations in the drug target Smo, the amplification of the downstream signaling, or the amplification of other interfering pathways (e.g.…”

Section: Discussionmentioning

confidence: 99%

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

Bánvölgyi

Anker

Lőrincz

et al. 2019

Journal of Dermatological Treatment

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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.

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Patient With Gorlin Syndrome and Metastatic Basal Cell Carcinoma Refractory to Smoothened Inhibitors

Cited by 14 publications

References 13 publications

Basal cell naevus syndrome: an update on genetics and treatment

Basal cell naevus syndrome: an update on genetics and treatment

Vismodegib for radiation-induced multiple basal cell carcinomas (BCCs) of the scalp

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

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