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Effective and Tolerable Treatment of Advanced Basal Cell Carcinoma With Vismodegib Despite Renal Insufficiency ONLINE FIRST

Lara Valeska Maul, MD1; Katharina C. Kähler, MD1; Axel Hauschild, MD1
[+] Author Affiliations
1Department of Dermatology, University Hospital Schleswig-Holstein, Campus Kiel, Kiel, Germany
JAMA Dermatol. Published online December 30, 2015. doi:10.1001/jamadermatol.2015.4592
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This case report describes treatment of advanced basal cell carcinoma with vismodegib in the context of renal insufficiency.

Recently, the hedgehog inhibitor vismodegib (Erivedge; Genentech) has demonstrated efficacy in the treatment of locally advanced and metastatic basal cell carcinoma (BCC).1,2 Almost all BCC contains genetic alterations in the sonic hedgehog signaling pathway. Most mutations affect the function of patched homologue 1, which normally inhibits the signaling activity of the transmembrane protein smoothened homologue. In a phase 1 study of vismodegib,1 a 58% response rate among 33 patients with advanced BCC was observed. The approval of vismodegib treatment at 150 mg/d was based on results from the phase 2 study.2 That study found the response rate for metastatic BCC to 30% and for locally advanced BCC, 43%, with a complete response in 21% of the cases. Common adverse events reported by more than 30% of patients were muscle spasms, alopecia, dysgeusia, weight loss, and fatigue.

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Vismodegib Treatment of Locally Advanced, Ulcerated Basal Cell Carcinoma of the Right Midface

A, Osseous and muscular tumor infiltration of both orbits and the nasal bridge before vismodegib treatment initiation. B, Conversion of secreting tissue into dry tumor tissue after 11 months of treatment with vismodegib.

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