Department of Hematology/Oncology, Children's Medical Center, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, China.
Guangdong Provincial Key Laboratory of Malignant Tumor Epigenetics and Gene Regulation, Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University, Guangzhou, China.
Pediatr Blood Cancer. 2024 Dec;71(12):e31340. doi: 10.1002/pbc.31340. Epub 2024 Sep 27.
Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis (EBV-HLH) is a type of pediatric HLH that occurs frequently in Asia. Although immunochemotherapy based on etoposide and hormone has improved survival rates, there are still about 30% of HLH patients that do not respond. The objective of the article is to examine the efficacy and safety of programmed cell death protein 1 (PD-1) inhibitors for children with relapsed/refractory (r/r) EBV-HLH.
A retrospective case note review of four pediatric patients with r/r EBV-HLH who were treated with PD-1 inhibitors at Sun Yat-sen Memorial Hospital, Sun Yat-sen University.
All four patients responded to PD-1 inhibitors and achieved partial response after their first infusion. Plasma EBV DNA copy number and HLH-related monitoring indicators decreased in all of these patients. All patients received allogeneic hematopoietic stem cell transplantation (allo-HSCT), and two were still alive at the last follow-up on December 30, 2022. Two patients died because of transplantation-related complications. Serious side effects included increased liver enzymes and edema in two patients.
PD-1 inhibitors are an effective salvage therapy and can provide a bridge to allo-HSCT for pediatric patients with r/r EBV-HLH. However, side effects should be monitered.
Epstein-Barr 病毒相关噬血细胞性淋巴组织细胞增生症（EBV-HLH）是一种常见于亚洲的儿科 HLH。尽管基于依托泊苷和激素的免疫化学疗法提高了生存率，但仍有约 30%的 HLH 患者没有反应。本文旨在研究程序性细胞死亡蛋白 1（PD-1）抑制剂对复发/难治性（r/r）EBV-HLH 儿童的疗效和安全性。
回顾性分析中山大学孙逸仙纪念医院收治的 4 例 r/r EBV-HLH 儿童接受 PD-1 抑制剂治疗的病例资料。
所有 4 例患者对 PD-1 抑制剂均有反应，首次输注后达到部分缓解。所有患者的血浆 EBV DNA 拷贝数和 HLH 相关监测指标均降低。所有患者均接受了异基因造血干细胞移植（allo-HSCT），截至 2022 年 12 月 30 日最后一次随访时，2 例仍存活。2 例患者因移植相关并发症死亡。严重的副作用包括 2 例患者的肝酶升高和水肿。
PD-1 抑制剂是一种有效的挽救治疗方法，可为 r/r EBV-HLH 儿童提供 allo-HSCT 的桥梁。但是，应该监测副作用。
