L-DEP方案治疗儿童慢性活动性EB病毒感染的疗效
Outcome of L-DEP regimen for treatment of pediatric chronic active Epstein-Barr virus infection.
作者信息
Ma Honghao, Zhang Liping, Wei Ang, Yang Jun, Wang Dong, Zhang Qing, Zhao Yunze, Chen Sitong, Lian Hongyun, Zhang Li, Zhou Chunju, Qin Maoquan, Li Zhigang, Wang Tianyou, Zhang Rui
机构信息
Hematology Center, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education; Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, 100045, China.
Hematologic Disease Laboratory, Hematology Center, Beijing Key Laboratory of Pediatric Hematology Oncology; National Key Discipline of Pediatrics (Capital Medical University); Key Laboratory of Major Diseases in Children, Ministry of Education; Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, 100045, China.
出版信息
Orphanet J Rare Dis. 2021 Jun 10;16(1):269. doi: 10.1186/s13023-021-01909-y.
PURPOSE
We intended to investigate the clinical features of paediatric patients with chronic active Epstein-Barr virus infection (CAEBV) and to examine the effectiveness of the L-DEP regimen before haematopoietic stem cell transplantation (HSCT).
METHODS
A retrospective analysis was performed on 35 patients with CAEBV at Beijing Children's Hospital from January 2016 to January 2020. The efficacy and adverse events of the L-DEP regimen were evaluated.
RESULTS
The median age of the 35 patients was 7.0 years old (range 2.5-17.5 years). Twenty-eight patients achieved a clinical response (80.0%, 22 in clinical CR, 6 in clinical PR) after L-DEP. In terms of virological response, 7 patients (20%) were assessed as having virological CR, and 23 patients (65.7%) had virological PR. Finally, 29 patients underwent allo-HSCT. The median survival time was 18 months (2-50 months). The 3-year overall survival rates in patients treated with chemotherapy only (n = 6) and chemotherapy followed by HSCT (n = 25) were 33.3% and 75.4%, respectively. After L-DEP 1st treatment and L-DEP 2nd treatment, the EBV-DNA loads in blood and plasma were significantly reduced compared with those before chemotherapy (median: 4.29 × 10 copies/ml vs. 1.84 × 10 copies/ml, Mann-Whitney U: P = 0.0004; 5.00 × 10 copies/ml vs. 3.17 × 10 copies/ml, Mann-Whitney U; P = 0.003; 2.27 × 10 copies/ml vs. 1.84 × 10 copies/ml, P = 0.0001; 5.00 × 10 copies/ml vs. 3.17 × 10 copies/ml, P = 0.003). Compared with the liver and spleen size before chemotherapy, the size of the liver and spleen shrank significantly after L-DEP 2nd (median 3.8 cm vs. 1.9 cm, P = 0.003; 3.8 cm vs. 0 cm, P < 0.008). In addition, after L-DEP treatment, there was no difference in the clinical or virological response rate regardless of HLH status (clinical response: 77.3% vs. 84.6%, P = 0.689; virological response: 90.9% vs. 76.9%, P = 0.337).
CONCLUSION
The L-DEP regimen is an effective therapy in CAEBV for bridging to allo-HSCT.
目的
我们旨在研究慢性活动性EB病毒感染（CAEBV）儿科患者的临床特征，并检验造血干细胞移植（HSCT）前L-DEP方案的有效性。
方法
对2016年1月至2020年1月在北京儿童医院就诊的35例CAEBV患者进行回顾性分析。评估L-DEP方案的疗效和不良事件。
结果
35例患者的中位年龄为7.0岁（范围2.5 - 17.5岁）。L-DEP治疗后，28例患者获得临床缓解（80.0%，22例临床完全缓解，6例临床部分缓解）。在病毒学反应方面，7例患者（20%）被评估为病毒学完全缓解，23例患者（65.7%）有病毒学部分缓解。最后，29例患者接受了异基因造血干细胞移植。中位生存时间为18个月（2 - 50个月）。单纯接受化疗的患者（n = 6）和化疗后接受HSCT的患者（n = 25）的3年总生存率分别为33.3%和75.4%。L-DEP第1次治疗和L-DEP第2次治疗后，血液和血浆中的EBV-DNA载量与化疗前相比显著降低（中位数：4.29×10拷贝/ml对1.84×10拷贝/ml，Mann-Whitney U检验：P = 0.0004；5.00×10拷贝/ml对3.17×10拷贝/ml，Mann-Whitney U检验；P = 0.003；2.27×10拷贝/ml对1.84×10拷贝/ml，P = 0.0001；5.00×10拷贝/ml对3.17×10拷贝/ml，P = 0.003）。与化疗前肝脏和脾脏大小相比，L-DEP第2次治疗后肝脏和脾脏大小显著缩小（中位数3.8 cm对1.9 cm，P = 0.003；3.8 cm对0 cm，P < 0.008）。此外，L-DEP治疗后，无论噬血细胞性淋巴组织细胞增生症（HLH）状态如何，临床或病毒学反应率均无差异（临床反应：77.3%对84.6%，P = 0.689；病毒学反应：90.9%对76.9%，P = 0.337）。
结论
L-DEP方案是CAEBV患者桥接异基因造血干细胞移植的有效治疗方法。
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