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

Oncologist. 2020 Dec;25(12):1001-1004. doi: 10.1002/onco.13541. Epub 2020 Oct 12.

Abstract

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.

Keywords: Dabrafenib; Histiocytosis; Targeted therapy; Vemurafenib.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Histiocytosis, Langerhans-Cell* / drug therapy
  • Histiocytosis, Langerhans-Cell* / genetics
  • Humans
  • Molecular Targeted Therapy
  • Mutation
  • Proto-Oncogene Proteins B-raf* / genetics
  • Retrospective Studies

Substances

  • BRAF protein, human
  • Proto-Oncogene Proteins B-raf