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

Antonious Z. Hazim, Gordon J. Ruan, Aishwarya Ravindran, Jithma P. Abeykoon, Caleb Scheckel, Robert Vassallo, Jay H. Ryu, W. Oliver Tobin, Matthew J. Koster, N. Nora Bennani, Karen L. Rech, Jason R. Young, Mithun V. Shah, Gaurav Goyal, Ronald S. Go

Research output: Contribution to journalComment/debatepeer-review

2 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)1001-1004
Number of pages4
Issue number12
StatePublished - Dec 2020


  • Dabrafenib
  • Histiocytosis
  • Targeted therapy
  • Vemurafenib

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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