Objective: To investigate the efficacy and safety of ruxolitinib, liposomal doxorubicin, etoposide, methylprednisolone+/-PEG-asparaginase (RU-DEP+/-L) in the treatment of relapsed/refractory (R/R) pediatric hemophagocytic lymphohistiocytosis (HLH). Methods: The clinical data of R/R pediatric HLH, who accepted the RU-DEP+/-L regimen at Beijing Children's Hospital from January 2018 to December 2019 was retrospectively analyzed. Results: A total of 16 patients were included in this study, including 13 males and 3 females, aged[M(Q1,Q3)] 1 (1, 2) years at diagnosis. Thirteen patients were diagnosed with Epstein-Barr virus (EBV)-HLH, 2 with EBV-induced primary HLH, and 1 with unclear etiology, among which 3 patients were co-infected with CMV. After the first-line treatment, 11 patients had no response, and 5 patients relapsed after complete response. Nine patients received the RU-L-DEP regimen, and 7 patients received the RU-DEP regimen. The overall response rate and complete response of RU-DEP+/-L treatment were 10/16 and 3/16, respectively. The negative conversion rate of plasma EBV-DNA was 7/15. The median follow-up time was 35.1 (2.4, 40.7) months, and 9/16 patients were survival. The 3-year overall survival rate after RU-DEP+/-L treatment in response and accepted hematopoietic stem cell transplantation (HSCT) was higher than that without response and did not receive HSCT (P=0.048). Among the 16 patients, 9 had varying degrees of myelosuppression, and 13 had an infection. Conclusions: RU-DEP+/-L can be used as a salvage treatment in R/R pediatric HLH, which can provide a bridge to HSCT and play an important role in the control of HLH. The main adverse reactions are myelosuppression and infection, which can be tolerated.
目的： 分析芦可替尼联合脂质体阿霉素、依托泊苷、甲泼尼龙和（或）培门冬酰胺酶（RU-DEP+/-L）对儿童复发/难治性（R/R）噬血细胞综合征［噬血细胞性淋巴组织细胞增多症（HLH）］的疗效以及安全性。 方法： 回顾性分析2018年1月至2019年12月期间于首都医科大学附属北京儿童医院血液病中心应用RU-DEP+/-L治疗的R/R HLH患儿。 结果： 共纳入16例患儿，男13例、女3例，诊断时年龄［M（Q1，Q3）］为1（1，2）岁。13例患儿诊断为EB病毒（EBV）-HLH，2例为EBV诱发的原发性HLH，1例为不明原因HLH，其中3例EBV-HLH同时合并巨细胞病毒（CMV）感染。一线治疗后，11例无反应，5例达到完全反应后再次复发。9例患儿采用了RU-L-DEP方案，7例患儿采用了RU-DEP方案。RU-DEP+/-L治疗总体反应率为10/16，完全反应率3/16，血浆EBV-DNA阴转率为7/15。随访时间35.1（2.4，40.7）个月，9/16患儿存活。RU-DEP+/-L治疗后获得反应衔接造血干细胞移植（HSCT）3年总生存率高于挽救治疗后无反应、未行HSCT患儿（P=0.048）。16例患儿中有9例出现不同程度的骨髓抑制，13例合并感染。 结论： RU-DEP+/-L可作为挽救治疗药物用于儿童R/R HLH的二线方案，可有效控制部分患儿HLH活动，为后续HSCT提供机会。不良反应主要是骨髓抑制及并发感染，患儿耐受可。.