BRAF 抑制剂
在几例复发性和难治性 LCH 以及其他组织细胞病（如 Erdheim-Chester 病，在成人中更常见）中，已报道对 BRAFV600E 抑制剂（如达拉非尼和维罗非尼）的无对照治疗反应。[109]Kieran MW, Cohen KJ, Doz F, et al. Complete radiographic responses in pediatric patients with BRAFV600E–positive tumors including highgrade gliomas: preliminary results of an ongoing phase 1/2a safety and pharmacokinetics (PK) study of dabrafenib. J Clin Oncol. 2014;32:5s(suppl; abstr 10056).http://meetinglibrary.asco.org/content/131336-144[110]Héritier S, Jehanne M, Leverger G, et al. Vemurafenib use in an infant for high-risk Langerhans cell histiocytosis. JAMA Oncol. 2015 Sep;1(6):836-8.http://oncology.jamanetwork.com/article.aspx?articleid=2279847http://www.ncbi.nlm.nih.gov/pubmed/26180941?tool=bestpractice.com[111]Donadieu J, Armari-Alla C, Templier I, et al. First use of vemurafenib in children LCH with neurodegenerative LCH. 30th Annual Histiocyte Society Meeting, Toronto; 2014.[112]Charles J, Beani JC, Fiandrino G, et al. Major response to vemurafenib in patient with severe cutaneous Langerhans cell histiocytosis harboring BRAF V600E mutation. J Am Acad Dermatol. 2014 Sep;71(3):e97-9.http://www.jaad.org/article/S0190-9622%2814%2901272-9/fulltexthttp://www.ncbi.nlm.nih.gov/pubmed/25128147?tool=bestpractice.com[113]Bubolz AM, Weissinger SE, Stenzinger A, et al. Potential clinical implications of BRAF mutations in histiocytic proliferations. Oncotarget. 2014 Jun 30;5(12):4060-70.http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4147306/http://www.ncbi.nlm.nih.gov/pubmed/24938183?tool=bestpractice.com 但是，耐药性的产生，以及使用单一抑制剂治疗 BRAFV600E 突变黑色素瘤存在的皮肤癌和胰腺癌潜在风险，这些均提示需要针对难治性或多次复发性 LCH 患者进行 BRAF 抑制剂前瞻性试验（单独使用或与此通路的其他抑制剂联用）。
