Efficacy of BRAF-Inhibitor Therapy in BRAFV600E-Mutated Adult Langerhans Cell Histiocytosis

Academic Article


  • Langerhans cell histiocytosis (LCH) is a rare histiocytic neoplasm. To date, there is a lack of U.S. Food and Drug Administration–approved treatments in adult LCH to establish optimal first-line therapy. We conducted a retrospective, single-center case series evaluating the use of BRAF inhibitors in adult patients with BRAFV600E- LCH proven by biopsy. Our case series is the first to report the use of BRAF inhibitors as first-line therapy in adults with LCH. We also report the efficacy with single-agent dabrafenib in adult LCH. All but one of our patients had favorable response to targeted therapy.
  • Digital Object Identifier (doi)

    Author List

  • Hazim AZ; Ruan GJ; Ravindran A; Abeykoon JP; Scheckel C; Vassallo R; Ryu JH; Tobin WO; Koster MJ; Bennani NN
  • Start Page

  • 1001
  • End Page

  • 1004
  • Volume

  • 25
  • Issue

  • 12