Hazim Antonious Z, Ruan Gordon J, Ravindran Aishwarya, Abeykoon Jithma P, Scheckel Caleb, Vassallo Robert, Ryu Jay H, Tobin W Oliver, Koster Matthew J, Bennani N Nora, Rech Karen L, Young Jason R, Shah Mithun V, Goyal Gaurav, Go Ronald S
Department of Internal Medicine, Mayo Clinic, Rochester, Minnesota, USA.
Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, Minnesota, USA.
Oncologist. 2020 Dec;25(12):1001-1004. doi: 10.1002/onco.13541. Epub 2020 Oct 12.
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 - 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.
朗格汉斯细胞组织细胞增多症（LCH）是一种罕见的组织细胞肿瘤。迄今为止，美国食品药品监督管理局尚未批准用于成人LCH的治疗方法来确立最佳一线治疗方案。我们进行了一项回顾性单中心病例系列研究，评估BRAF抑制剂在经活检证实为BRAF-LCH的成年患者中的应用。我们的病例系列是首个报道将BRAF抑制剂作为成人LCH一线治疗方法的研究。我们还报告了单药达拉非尼在成人LCH中的疗效。除一名患者外，我们所有患者对靶向治疗均有良好反应。
