Clinical efficacy of Ruxolitinib in combination with Methylprednisolone as a bridge to allogeneic hematopoietic stem cell transplantation for relapse/refractory Epstein-Barr virus-associated hemophagocytic syndrome in pediatric patients
10.3760/cma.j.cn101070-20200308-00343
- VernacularTitle:芦可替尼联合甲泼尼龙桥接异基因造血干细胞移植治疗儿童复发/难治性EB病毒相关性噬血细胞综合征的疗效
- Author:
Dao WANG
1
;
Yanjie DING
;
Jiao CHEN
;
Hongliang YOU
;
Huanhuan LI
;
Bai LI
;
Qianghua YAO
;
Yingchao WANG
;
Dingming WAN
;
Yufeng LIU
Author Information
1. 郑州大学第一附属医院儿科 450052
- Keywords:
Ruxolitinib;
Allo-hematopoietic stem cell transplantation;
Child;
Epstein-Barr virus;
Relapse/refractory hemophagocytic syndrome
- From:
Chinese Journal of Applied Clinical Pediatrics
2021;36(15):1185-1187
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To explore the clinical efficacy and safety of Ruxolitinib, a Janus kinase inhibitor, in combination with Methylprednisolone as a bridge to allogeneic hematopoietic stem cell transplantation (allo-HSCT) for relapsed/refractory Epstein-Barr virus-associated hemophagocytic syndrome (EBV-AHS) in pediatric patients.Methods:The clinical data of 4 patients with relapsed/refractory EBV-AHS treated with Ruxolitinib in combination with Methylprednisolone as a bridge to allo-HSCT at the Department of Pediatrics, the First Affiliated Hospital of Zhengzhou University from August 2018 to February 2020 were retrospectively analyzed, and the disease characteristics, diagnosis and treatment process, clinical experience and related research progress were analyzed and summarized.Results:Among 4 patients with relapsed/refractory EBV-AHS, 2 patients were treated with low-dose Ruxolitinb in combination with Methylprednisolone for 6-10 weeks after partial remission.The disease did not progress, and they survived after being bridged to allo-HSCT.One patient was treated with large-dose Ruxolitinib in combination with Methylprednisolone due to the intolerance to chemotherapy, with the biochemical indicators of hemophagocytic syndrome significantly improved, and then the bridging to allo-HSCT was performed 2 months ago and this patient survived.One patient with EBV-AHS relapsed was relieved by chemotherapy again, then was given maintenance therapy with Ruxolitinib and Methylprednisolone, but the condition still progressed and the treatment was ineffective.This patient underwent allo-HSCT for salvage treatment more than 1 year ago and survived.Except that 1 patient developed mild anemia, the other 3 patients had no significant Ruxolitinib-related toxicities.Conclusions:Ruxolitinib in combination with Methylprednisolone can be safely employed as a salvage treatment for pediatric patients with relapsed/refractory EBV-AHS and a bridge to allo-HSCT, which has favorable safety, efficacy and tolerance in clinical practice.
