芦可替尼治疗儿童噬血细胞性淋巴组织细胞增多症临床分析

Clinical analysis of ruxolitinib in the treatment of hemophagocytic lymphohistiocytosis in children

目的探讨应用Janus激酶(JAK1/JAK2)抑制剂芦可替尼治疗儿童噬血细胞性淋巴组织细胞增多症(hemophagocyticlymphohistiocytosis,HLH)的疗效及安全性。方法回顾性分析2018年1月至2018年6月于首都儿科研究所附属儿童医院血液科诊治的2例经常规治疗无效、在监护人知情同意后予芦可替尼口服治疗病情缓解的HLH患儿的临床资料,并结合相关文献复习芦可替尼作为靶向药物治疗HLH的作用机制及临床效果。结果 2例HLH患儿应用芦可替尼治疗后临床症状缓解,血象、肿瘤坏死因子α、白细胞介素2受体、铁蛋白及纤维蛋白原等指标好转,治疗期间疾病无进展,未发生严重药物不良反应。结论儿童HLH是一种病情凶险且死亡率极高的疾病,少部分患儿对于传统化疗无反应或不能耐受,早期应用Janus激酶抑制剂芦可替尼可通过减轻细胞因子引发的炎症反应以干预疾病进展,安全有效,耐受性良好。

Objective To investigate the efficacy and security of janus kinase inhibitor ruxolitinib in the treatment of hemophagocytic lymphohistiocytosis in children. Method 2 cases patients with HLH were analyzed retrospectively who were not treated with routine therapy but had remission after treatment with ruxolitinib. Combined with the relevant literature, reviewing the principle and clinical ef? cacy of Janus kinase inhibitor ruxolitinib, as a targeted drug for the treatment of HLH. Result After orally treated with ruxolitinib,two patients received clinical remission, the laboratory indexes were improved, the disease did not progress during the treatment, and without any serious adverse drug reaction. Conclusion HLH is a dangerous disease in children that with a high mortality rate. A small number of patients have no response to the routine therapy. So early application of ruxolitinib to intervene the progression of HLH by reducing the in? ammatory response induced by cytokines is safe, effective and well tolerated.