Luo Yanhui, Wei Ang, Wang Bin, Zhu Guanghua, Zhang Rui, Jia Chenguang, Yan Yan, Zhou Xuan, Yang Jun, Qin Maoquan, Wang Tianyou
Department of Stem cell Transplantation, 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 China.
Pediatr Investig. 2022 Nov 15;6(4):250-259. doi: 10.1002/ped4.12350. eCollection 2022 Dec.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is considered the only effective treatment for chronic active Epstein-Barr virus infection (CAEBV). The clinical efficacy and safety of allo-HSCT with different conditioning regimens in children with CAEBV remain unclear.
To evaluate the effectiveness and safety of allo-HSCT with the modified myeloablative conditioning (MAC) regimen for children with CAEBV and also the factors affecting the outcomes.
We retrospectively analyzed children with CAEBV who underwent allo-HSCT with the modified MAC regimen at Beijing Children's Hospital, Capital Medical University from October 2016 to June 2021. Data related to the clinical manifestations, engraftment, and outcome were extracted from the medical records.
The cohort comprised 41 patients (24 males, 17 females) with a median transplantation age of 92.6 (60.4, 120.7) months and a median follow-up time of 28.2 (15.3, 40.2) months. Four patients (9.8%) died, among which three died from primary disease relapse, and one died from grade IV acute graft-versus-host diseases (aGVHD) after stopping treatment. The 3-year overall survival (OS) and 3-year event-free survival (EFS) rates were 88.8% ± 5.4% and 85.0% ± 5.7%, respectively. The 3-year OS and EFS did not significantly differ between the patients with hemophagocytic lymphohistiocytosis (HLH) and the patient without HLH (87.7% ± 6.8% . 91.7% ± 8.0%, = 0.790; 85.0% ± 6.9% . 84.6% ± 10.0%, = 0.921), or among the patients with complete remission, partial remission, and activity disease before HSCT (all > 0.05). Multivariate analysis showed that grade III-IV aGVHD was a risk factor for mortality (Hazards ratio: 11.65, 95% confidence interval: 1.00, 136.06; = 0.050).
Allo-HSCT with the modified MAC regimen is safe and effective for pediatric CAEBV. This treatment benefits patients with HLH or active disease. Patients with Grade III-IV aGVHD may be associated with worse outcomes.
异基因造血干细胞移植（allo-HSCT）被认为是慢性活动性EB病毒感染（CAEBV）的唯一有效治疗方法。不同预处理方案的allo-HSCT治疗儿童CAEBV的临床疗效和安全性尚不清楚。
评估改良清髓预处理（MAC）方案的allo-HSCT治疗儿童CAEBV的有效性和安全性，以及影响预后的因素。
我们回顾性分析了2016年10月至2021年6月在首都医科大学附属北京儿童医院接受改良MAC方案allo-HSCT的CAEBV患儿。从病历中提取有关临床表现、植入情况和预后的数据。
该队列包括41例患者（男24例，女17例），移植年龄中位数为92.6（60.4，120.7）个月，中位随访时间为28.2（15.3，40.2）个月。4例患者（9.8%）死亡，其中3例死于原发病复发，1例在停止治疗后死于IV级急性移植物抗宿主病（aGVHD）。3年总生存率（OS）和3年无事件生存率（EFS）分别为88.8%±5.4%和85.0%±5.7%。噬血细胞性淋巴组织细胞增生症（HLH）患者和非HLH患者的3年OS和EFS无显著差异（87.7%±6.8% 对91.7%±8.0%，P = 0.790；85.0%±6.9% 对84.6%±10.0%，P = 0.921），在HSCT前完全缓解、部分缓解和疾病活动的患者中也无显著差异（均P>0.05）。多因素分析显示，III-IV级aGVHD是死亡的危险因素（风险比：11.65，95%置信区间：1.00，136.06；P = 0.050）。
改良MAC方案的allo-HSCT治疗儿童CAEBV安全有效。这种治疗方法对HLH或疾病活动的患者有益。III-IV级aGVHD患者的预后可能较差。
