[4名儿童因感染 Epstein-Barr 病毒而引发的难治性原发性嗜血细胞淋巴组织细胞增多症的 L-DEP 方案挽救疗法]。

Y. Z. Zhao, H. H. Ma, H. Lian, D. Wang, T. Y. Wang, R. Zhang
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引用次数: 0

摘要

目的分析L-DEP方案(天冬酰胺酶、脂质体多柔比星、依托泊苷和甲基强的松龙)作为一种挽救疗法治疗由爱泼斯坦-巴氏病毒感染引发的难治性原发性嗜血细胞淋巴组织细胞增多症(EBV-pHLH)的有效性和安全性。研究方法在这项回顾性病例研究中,收集了2016年1月至2022年6月期间在北京儿童医院确诊的4例EBV-pHLH患儿接受L-DEP方案治疗前后的临床和实验室数据,分析了L-DEP方案治疗EBV-pHLH的疗效和安全性。结果4例患者中,女性3例,男性1例,年龄在0.8岁至7.0岁之间。其中2例为PRF1复合杂合突变,1例为UNC13D杂合突变,1例为ITK同源突变。在接受 L-DEP 治疗前,所有患者均有贫血和可溶性 CD25 水平飙升,3 名患者有中性粒细胞减少和血栓性血小板减少,3 名患者铁蛋白水平较高,3 名患者有低纤维蛋白原血症,1 名患者有高甘油三酯血症。在接受 1 或 2 个周期的 L-DEP 治疗后,3 名患者的病情得到缓解,包括完全缓解(1 例)和部分缓解(2 例),另一名患者的病情没有缓解。3 名获得缓解的患者的血细胞计数、可溶性 CD25、甘油三酯、纤维蛋白原和白蛋白水平逐渐恢复。四名患者在接受 L-DEP 治疗后都进行了造血干细胞移植(HSCT),其中三人存活。所有患者均未出现严重的化疗相关并发症。主要的副作用是骨髓抑制、感染和胰腺炎,这些副作用在经过适当治疗后都已恢复,只有一名患者在紧急造血干细胞移植后死于严重感染。结论L-DEP方案可作为难治性小儿EBV-pHLH有效、安全的挽救治疗方案,同时也为患者提供了接受造血干细胞移植的机会。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
[L-DEP regimen salvage therapy for refractory primary hemophagocytic lymphohistiocytosis triggered by Epstein-Barr virus infection in 4 children].
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.
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