丝裂原活化蛋白激酶（MAPK）通路的靶向抑制：进展性危及生命的多系统朗格汉斯细胞组织细胞增多症新出现的挽救治疗选择
Targeted inhibition of the MAPK pathway: emerging salvage option for progressive life-threatening multisystem LCH.
作者信息
Kolenová Alexandra, Schwentner Raphaela, Jug Gunhild, Simonitsch-Klupp Ingrid, Kornauth Christoph, Plank Lukáš, Horáková Júlia, Bodová Ivana, Sýkora Tomáš, Geczová Lucia, Holter Wolfgang, Minkov Milen, Hutter Caroline
机构信息
Department of Pediatric Hematology and Oncology, Comenius University Medical School, Bratislava, Slovakia.
University Children's Hospital, Bratislava, Slovakia.
出版信息
Blood Adv. 2017 Feb 2;1(6):352-356. doi: 10.1182/bloodadvances.2016003533. eCollection 2017 Feb 14.
Abstract
Single-agent vemurafenib leads to a rapid and sustained clinical response in severe multisystem LCH but does not eradicate the disease.Longitudinal assessment of V600E during treatment shows that clinical remission can occur despite significant amounts of mutated BRAF.
摘要
单药威罗菲尼可使严重多系统朗格汉斯细胞组织细胞增多症（LCH）产生快速且持续的临床反应，但无法根除该疾病。治疗期间对V600E进行纵向评估显示，尽管存在大量BRAF基因突变，仍可出现临床缓解。
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