应用索拉非尼单药治疗复发难治性朗格汉斯细胞组织细胞增生症2例及文献复习被引量：4

Application of sorafenib monotherapy in the treatment of relapsed and refractory Langerhans cell histiocytosis: a report of 2 cases and literature review

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摘要目的通过报道2例复发难治性朗格汉斯细胞组织细胞增生症（langerhans cell histiocytosis,LCH）,总结其临床特征,以期提高临床医生对BRAFV600E基因突变的检查及靶向治疗的认识。方法回顾性分析2014年10~11月本院血液科收治的2例应用索拉非尼[150mg/（m2·d）,每天2次]单药口服治疗后症状缓解、病情得到控制的复发难治性LCH患儿的资料,结合相关文献,复习BRAFV600E基因在LCH患者中的突变情况、索拉非尼作为靶向药物治疗的原理。结果 2例BRAFV600E阳性的LCH患者应用靶向药物索拉非尼单药口服后,临床症状缓解。化验指标好转,未发生严重药物不良反应。结论索拉非尼药物治疗外周血BRAFV600E基因突变阳性的LCH患者,安全有效。本文报道例数少,尚需行进一步的大样本治观察,以进一步验证疗效。需要长期随访应用索拉非尼口服治疗患者,观察不良反应,同时确定服药时间及停药标准。 Objective Through the report about two cases of patients with persistent or recurrent langerhans cell histiocytosis（LCH）, to summiarize the clinical characteristics, in order to improve clinicians of BRAFV600 E mutation screening and targeted therapy. Method 2 cases patients with LCH were analyzed retrospectively which treated by sorafenib monotherapy [150mg/（m2·d）, 2 times a day],and symptoms remission get control of relapsed and refractory, combined with the relevant literature, the BRAFV600 E gene mutations in patients with LCH mutation and sorafenib as targeted drug therapy principle were reviewed. Result After orally treated with sorafenib alone, the clincal manifestions of two patients with oncogenic BRAF-V600 E mutation receive clinical remission and without any serious adverse drug reactions. Conclusion Treating with sorafenib in patients with oncogenic BRAF-V600 E mutations in peripheral blood is a safe and potent approach. Since the number of sample in this paper is small, it is necessary for us to take a further study to verify its efficacy. Long term follow-up is needed for obeservation of adverse reactions and determining the optimal treatment courses and its stopping standards.

作者曹静刘嵘李君惠师晓东罗丹青胡涛冯顺乔张朝霞邵端芳

机构地区首都儿科研究所附属儿童医院

出处《中国医刊》 CAS 2016年第7期71-74,共4页 Chinese Journal of Medicine

关键词朗格汉斯细胞组织细胞增生症 BRAFV600E 索拉非尼 Langerhans cell histiocytosis BRAFV600E Sorafenib

分类号 R551.1 [医药卫生—血液循环系统疾病]

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