Objective: To analyze the efficacy and safety of the L-DEP regimen (asparaginase, liposome doxorubicin, etoposide and methylprednisolone) as a salvage therapy for the refractory primary hemophagocytic lymphohistocytosis triggered by Epstein-Barr virus infection (EBV-pHLH) in children. Methods: In this retrospective case study, clinical and laboratory data before and after L-DEP regimen of 4 children diagnosed with EBV-pHLH in Beijing Children's hospital between January 2016 and June 2022 were collected, and the efficacy and safety of L-DEP regimen for the treatment of EBV-pHLH were analyzed. Results: Among 4 patients, there were 3 females and 1 male with the age ranged from 0.8 to 7.0 years. Two of them showed compound heterozygous mutations of PRF1, one with a heterozygous mutation of UNC13D, one homozygous mutation of ITK. Before the L-DEP therapy, all of them had anemia and a soaring level of soluble CD25, 3 patients had neutropenia and thrombopenia, 3 patients had a high level of ferritin, 3 patients had hypofibrinogenemia and 1 patient had hypertriglyceridemia. After receiving 1 or 2 cycles of L-DEP treatment, three achieved remission, including complete remission (1 case) and partial remission (2 cases), and the other one had no remission. The levels of blood cell counts, soluble CD25, triglyceride, fibrinogen and albumin were recovered gradually in 3 patients who got remission. All four patients underwent hematopoietic stem cell transplantation (HSCT) after L-DEP regimen, and three survived. All patients had no severe chemotherapy related complications. The main side effects were bone marrow suppression, infection and pancreatitis, which recovered after appropriate treatments, apart from one who died from severe infection after urgent HSCT. Conclusion: L-DEP regimen could be served as an effective and safe salvage treatment for refractory pediatric EBV-pHLH, and also provide an opportunity for patients to receive HSCT.
目的： 观察门冬酰胺酶、脂质体阿霉素、依托泊苷和甲泼尼龙（L-DEP）方案挽救治疗难治性EB病毒感染诱发的原发性噬血细胞综合征（EBV-pHLH）的有效性和安全性。 方法： 回顾性病例总结。以2016年1月至2022年6月就诊于首都医科大学附属北京儿童医院的4例难治性EBV-pHLH患儿为研究对象，收集L-DEP方案治疗前后的临床及实验室资料，分析L-DEP方案治疗EBV-pHLH患儿的有效性及安全性。 结果： 4例患儿中女3例、男1例，年龄0.8~7.0岁，PRF1基因复合杂合突变2例，UNC13D基因杂合突变1例，ITK基因纯合突变1例。在L-DEP方案化疗前，所有患儿均有贫血和可溶性CD25升高，3例存在中性粒细胞和血小板计数减少，3例铁蛋白水平升高，3例纤维蛋白原水平下降，1例甘油三酯水平明显升高。在接受1~2个疗程L-DEP方案化疗后，3例获得缓解，包括完全缓解（1例）和部分缓解（2例），另1例未缓解。3例缓解患儿血细胞计数、可溶性CD25、甘油三酯、纤维蛋白原、白蛋白水平均有所恢复。4例患儿均在完成L-DEP方案化疗后接受造血干细胞移植，其中长期生存3例。4例患儿均未出现严重的化疗不良反应，主要不良反应为骨髓抑制、感染、胰腺炎，除1例接受紧急移植患儿死于重症感染外，另3例患儿均存活。 结论： L-DEP方案可以作为EBV-pHLH患儿安全有效的挽救治疗方案，为患儿顺利接受造血干细胞移植提供宝贵机会。.