title: Pivotal ERIVANCE basal cell carcinoma (BCC) study: 12-month update of efficacy and safety of vismodegib in advanced BCC.
abstract: BACKGROUND
  Primary analysis from the pivotal ERIVANCE BCC study resulted in approval of vismodegib, a Hedgehog pathway inhibitor indicated for treatment of adults with metastatic or locally advanced basal cell carcinoma (BCC) that has recurred after surgery or for patients who are not candidates for surgery or radiation.
  
  
  OBJECTIVE
  An efficacy and safety analysis was conducted 12 months after primary analysis.
  
  
  METHODS
  This was a multinational, multicenter, nonrandomized, 2-cohort study in patients with measurable and histologically confirmed locally advanced or metastatic BCC taking oral vismodegib (150 mg/d). Primary outcome measure was objective response rate (complete and partial responses) assessed by independent review facility.
  
  
  RESULTS
  After 12 months of additional follow-up, median duration of exposure to vismodegib was 12.9 months. Objective response rate increased from 30.3% to 33.3% in patients with metastatic disease, and from 42.9% to 47.6% in patients with the locally advanced form. Median duration of response in patients with locally advanced BCC increased from 7.6 to 9.5 months. No new safety signals emerged with extended treatment duration.
  
  
  CONCLUSIONS
  Limitations include low prevalence of advanced BCC and challenges of designing a study with heterogenous manifestations.
  
  
  CONCLUSIONS
  The 12-month update of the study confirms the efficacy and safety of vismodegib in management of advanced BCC.
authors.affiliation: Mayo Clinic, Scottsdale, Arizona. Electronic address: sekulic.aleksandar@mayo.edu.
authors.forename: Aleksandar
authors.initials: A
authors.lastname: Sekulic
authors.affiliation: University of Texas MD Anderson Cancer Center, Houston, Texas.
authors.forename: Michael R
authors.initials: MR
authors.lastname: Migden
authors.affiliation: University of Colorado Cancer Center, Denver, Colorado.
authors.forename: Karl
authors.initials: K
authors.lastname: Lewis
authors.affiliation: Sarah Cannon Research Institute, Nashville, Tennessee.
authors.forename: John D
authors.initials: JD
authors.lastname: Hainsworth
authors.affiliation: Ameriderm Research, Ormond Beach, Florida; University of Central Florida, Orlando, Florida; University of Illinois, Urbana, Illinois.
authors.forename: James A
authors.initials: JA
authors.lastname: Solomon
authors.affiliation: Northwestern University, Evanston, Illinois.
authors.forename: Simon
authors.initials: S
authors.lastname: Yoo
authors.affiliation: University of California, San Francisco, San Francisco, California.
authors.forename: Sarah T
authors.initials: ST
authors.lastname: Arron
authors.affiliation: Dana-Farber Cancer Institute, Boston, Massachusetts; Mount Sinai Medical Center, New York, New York.
authors.forename: Philip A
authors.initials: PA
authors.lastname: Friedlander
authors.affiliation: Mount Sinai Medical Center, New York, New York.
authors.forename: Ellen
authors.initials: E
authors.lastname: Marmur
authors.affiliation: Johns Hopkins University, Baltimore, Maryland.
authors.forename: Charles M
authors.initials: CM
authors.lastname: Rudin
authors.affiliation: Stanford University School of Medicine, Palo Alto, California.
authors.forename: Anne Lynn S
authors.initials: AL
authors.lastname: Chang
authors.affiliation: Sint-Augustinus Hospital, Antwerp, Belgium.
authors.forename: Luc
authors.initials: L
authors.lastname: Dirix
authors.affiliation: Genentech Inc, South San Francisco, California.
authors.forename: Jeannie
authors.initials: J
authors.lastname: Hou
authors.affiliation: Genentech Inc, South San Francisco, California.
authors.forename: Huibin
authors.initials: H
authors.lastname: Yue
authors.affiliation: Universitätsklinikum Schleswig-Holstein, Kiel, Germany.
authors.forename: Axel
authors.initials: A
authors.lastname: Hauschild
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000813:Anilides / Q000627:therapeutic use*; D002280:Basal Cell Carcinoma / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology*; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D020714:Maximum Tolerated Dose; D008875:Middle Aged; D009361:Neoplasm Invasiveness / Q000473:pathology; D009367:Neoplasm Staging; D061214:Patient Safety / Q000706:statistics & numerical data; D011725:Pyridines / Q000627:therapeutic use*; D018570:Risk Assessment; D012878:Skin Neoplasms / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology*; D016019:Survival Analysis; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D000813:Anilides; C538724:HhAntag691; D011725:Pyridines
keywords: ERIVANCE BCC study; Hedgehog pathway inhibitor; advanced basal cell carcinoma; locally advanced basal cell carcinoma; metastatic basal cell carcinoma; vismodegib