Efficacy of BRAF-Inhibitor Therapy in <i>BRAF</i> <i>V600E</i>-Mutated Adult Langerhans Cell Histiocytosis
Antonious Hazim, Gordon 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 Vinod Shah, Gaurav Goyal, Ronald S. Go
Abstract
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.