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June 2017

Clinical Response to Pazopanib in a Patient With KDR-Mutated Metastatic Basal Cell Carcinoma

Author Affiliations
  • 1DeBartolo Family Personalized Medicine Institute, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida
  • 2Melanoma and Cutaneous Oncology Program, The Angeles Clinic and Research Institute, Los Angeles, California
  • 3Georgetown-Lombardi Comprehensive Cancer Center, Washington, DC
  • 4Department of Oncological Sciences, University of South Florida, Tampa, Florida
  • 5Head and Neck and Endocrine Oncology, H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida
JAMA Dermatol. 2017;153(6):607-609. doi:10.1001/jamadermatol.2017.0187

Next-generation sequencing (NGS) panels are increasingly used to identify genomic alterations that extend therapeutic opportunities for patients who progress beyond therapies approved by the US Food and Drug Administration yet remain candidates for treatment.1,2 Metastatic basal cell carcinoma (mBCC) is a deadly manifestation of the most commonly diagnosed skin cancer, with a mutational landscape primarily focused on the sonic hedgehog pathway (SHH), including patched homologue 1 (PTCH1) and smoothened homologue (SMO).3 Unfortunately once progression or therapy intolerance occurs, therapeutic options are limited. Thus genomic profiling of mBCC has emerged as a critical component of disease management. We report a case of a patient with mBCC who experienced a 9-month complete metabolic response (CR) after treatment with pazopanib, an agent targeting a kinase insert domain receptor (KDR) mutation.