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Arthika Chandramohan, Archana Nair, Anne L Chang, Andrea Kossler; Hedgehog Pathway Inhibitors as Neoadjuvant Therapy for Orbital/Periorbital Basal Cell Carcinoma. Invest. Ophthalmol. Vis. Sci. 2018;59(9):5621.
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To report our results using the Hedgehog Pathway Inhibitors (HPI) as neoadjuvant therapy for orbital and periorbital basal cell carcinoma (BCC)
This retrospective cohort study examined all patients who received some form of HPI therapy at Stanford Hospital Center between January 2010 and December 2016. Data were abstracted via clinical charts, imaging files, clinical photographs, and clinical study data. Primary outcome measures included total treatment time, resistance to treatment, time to regrowth after the end of HPI treatment. Self-reported side effect data was also collected for all patients.
Thirteen patients were identified: 12 treated with vismodegib and one with sonidegib. All patients were treated neoadjuvantly with the intent of post-HPI definitive surgical management. Median treatment length was 12 months (range 3.5-60 mos, SD 15.01 mos). Six patients had complete clinical response, six had only partial, and one demonstrated progression as defined by RECIST criteria. Four (66%) of those with complete responses had regrowth; the median time to regrowth from stopping HPI was 11.5 months (range 4-21 mos). These were all successfully treated with surgical excision. Four (30%) patients developed resistance during the first year of treatment. Median follow up duration after stopping HPI was 16.5 months (0.5 to 74 months) with three patients receiving continued therapy. Overall 92% (12) of patients had a partial or complete response to treatment. All but one patient reported either muscle spasms, alopecia, or dysgeusia as side effects of the HPI.
HPIs are effective neoadjuvant treatment for orbital or extensive periorbital BCC for patients needing exenteration disfiguring surgery or are poor surgical candidates. The majority of our patients had a partial or complete clinical response however 66% of patients with a complete reponse had regrowth after about a year. We believe HPIs have a role in neoadjuvant therapy in poor surgical candidates, however due to resistance and regrowth it is important to monitor these patients. We recommend adhering to FDA guidlines for HPI treatment until fruther studies can confirm these results.
This is an abstract that was submitted for the 2018 ARVO Annual Meeting, held in Honolulu, Hawaii, April 29 - May 3, 2018.
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