Posted: Wednesday, March 13, 2024
According to Christopher A. Barker, MD, of Memorial Sloan Kettering Cancer Center, New York, and colleagues, patients with locally advanced, unresectable basal cell carcinoma of the head and neck demonstrated high rates of locoregional tumor control and progression-free survival, as well as durable improvement in patient-reported quality of life, from the combination of the Hedgehog pathway inhibitor vismodegib with curative-intent radiation therapy. The results of their multicenter phase II trial, which were presented during the 2024 Multidisciplinary Head and Neck Cancers Symposium (Abstract 9), support the use of this combined-modality approach in clinical practice.
Patients with lesions of at least 2 cm and/or with nodal metastasis but without distant metastasis underwent induction therapy with vismodegib followed by concurrent vismodegib and radiation therapy. Of the 24 patients who received vismodegib, 5 were unable to complete induction therapy.
A total of 91% of patients achieved locoregional tumor control at 1 year. The response rate was 63% after induction therapy and 83% following the combined therapy. The progression-free and overall survival rates were 100% and 96% at 1 year and 78% and 83% at 5 years, respectively, with a median follow-up of 5.7 years. Distant metastases and basal cell carcinoma–related deaths were not observed.
The most frequently reported treatment-related adverse events were dysgeusia (83%), fatigue (75%), and myalgias (75%). Although seven serious adverse events were documented in four patients, none were deemed related to the study treatment. The investigators reported no grade 4 or 5 treatment-related adverse events.
Cinically meaningful benefits in patient-reported quality of life were demonstrated in all subscales, with improvements in both emotions and functioning persisting for a year after the end of treatment. All patients who underwent tumor sequencing appeared to harbor an oncogenic loss of function mutation in PTCH1; however, no functional alterations in SMO, SUFU, or GLI1 were detected.
Disclosure: For full disclosures of the study authors, visit astro.org.
2024 Multidisciplinary Head and Neck Cancers Symposium