Sonic Hedgehog Pathway Inhibitors: from Clinical Trials to Clinical Practice

Abstract

Basal cell carcinoma (BCC) is the most common malignancy. It can rarely become locally advanced (laBCC) or metastasize (mBCC). For recurrent, inoperable, laBCC and/or mBCC, Sonic Hedgehog (Shh) pathway inhibitors, vismodegib and sonidegib, are treatment options. This case series evaluates limitations surrounding their use in clinical practice.

This was an IRB‐approved retrospective case series of BCC treatment with vismodegib and/or sonidegib at our institution between January 2006 and March 2017, with follow‐ up through August 2018. Patients receiving concurrent Shh inhibitors and radiotherapy were excluded. The examined outcomes included clinical response, progression free survival (PFS), drug holidays (DH), and adverse effects (AE).

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