L-DEP regimen salvage therapy for paediatric patients with refractory Epstein-Barr virus-associated haemophagocytic lymphohistiocytosis
L-DEP regimen salvage therapy for paediatric patients with refractory Epstein-Barr virus-associated haemophagocytic lymphohistiocytosis
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L-DEP 方案对难治性 Epstein-Barr 病毒相关噬血细胞性淋巴组织细胞增多症儿科患者的挽救治疗
DOI：
10.1111/bjh.16861
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发表时间：
2020-06-11
影响因子：
6.5
通讯作者：
Zhang, Rui
中科院分区：
文献类型：
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作者：
Zhao, Yunze;Li, Zhigang;Zhang, Rui
EB病毒相关噬血细胞性淋巴组织细胞增生症（EBV-HLH）是继发性HLH最常见的亚型之一。然而，超过30%的患者对传统治疗没有反应。在此，我们研究了L-DEP方案作为儿科难治性EBV-HLH的挽救治疗的有效性和安全性。我们回顾性分析了2016年1月1日至2019年3月31日在北京儿童医院接受L-DEP方案治疗的26例难治性EBV-HLH儿童患者。5例患者达到完全缓解（CR），11例达到部分缓解（PR），表明总缓解率为61中心点5%（CR + PR）。在16名达到CR或PR的患者中，有10名接受了异基因造血干细胞移植（allo-HSCT），7名在2020年4月30日的末次随访时仍然存活。10例无反应的患者中有2例在allo-HSCT后存活。主要副作用包括淀粉酶升高、骨髓抑制和凝血障碍，尽管在大多数情况下这些可以通过支持治疗来控制。因此，我们得出结论，L-DEP方案是一种有效的和相对安全的挽救治疗儿童难治性EBV-HLH。该方案还为allo-HSCT提供了桥梁。
Epstein-Barr virus-associated haemophagocytic lymphohistiocytosis (EBV-HLH) is one of the most common subtypes of secondary HLH. However, more than 30% of patients do not respond to traditional treatment. Here, we investigated the efficacy and safety of the L-DEP regimen as a salvage therapy for paediatric refractory EBV-HLH. We retrospectively analysed 26 paediatric patients with refractory EBV-HLH who received the L-DEP regimen at Beijing Children's Hospital from 1 January 2016 to 31 March 2019. Five of the patients achieved complete response (CR) and 11 achieved partial response (PR), indicating an overall response rate of 61 center dot 5% (CR + PR). Ten of the 16 patients who achieved CR or PR received allogenic haematopoietic stem cell transplantation (allo-HSCT), and seven were still alive at the last follow-up on 30 April 2020. Two of the 10 patients who did not respond were alive after allo-HSCT. Major side effects included increased amylase, bone marrow suppression and coagulation disorders, though these could be controlled by supportive therapy in most cases. Thus, we conclude that the L-DEP regimen is an effective and relatively safe salvage therapy for children with refractory EBV-HLH. This regimen also provides a bridge to allo-HSCT.