Efficacy of BRAF-Inhibitor Therapy in BRAF V600E -Mutated Adult Langerhans Cell Histiocytosis

[摘要] 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 BRAF V600E - 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.

[学科分类] 地质学

[关键词] Vemurafenib;Dabrafenib;Targeted therapy;Histiocytosis