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Pediatric Drugs
Published in: Pediatric Drugs 5/2023

07-06-2023 | Ruxolitinib | Original Research Article

Ruxolitinib Treatment of Steroid-Refractory Graft-versus-Host Disease in Children: A Case Series and Review of the Literature

Authors: Wei-Ling Yan, Fen-Ying Zhao, Min-Er Gu, Nan Liu, Xiao-Ping Guo, Xiao-Jun Xu

Published in: Pediatric Drugs | Issue 5/2023

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Abstract

Background

Ruxolitinib has been increasingly used in the treatment of steroid-refractory graft-versus-host disease (SR-GVHD) in allogeneic hematopoietic stem cell transplantation (allo-HSCT) patients. However, there are limited data on the use of ruxolitinib in children.

Objective

This study aimed to assess the efficacy and toxicity of ruxolitinib in the treatment of SR-GVHD in children.

Patients and Methods

Data of patients who suffered from SR-GVHD after allo-HSCT and received ruxolitinib treatment between June 2018 and December 2020 at our center were analyzed retrospectively. The characteristics of patients, the dosage of ruxolitinib, the response, toxicity, and the survival data were collected.

Results

A total of 14 pediatric patients were diagnosed with SR-GVHD after allo-HSCT and received ruxolitinib. The age of the patients ranged from 3 months to 12 years old. The dosage of ruxolitinib ranged from 2.5 mg twice daily to 7.5 mg twice daily, mainly according to patient weight. The total overall response rate (ORR) was 64.3% (9/14), with 63.6% (7/11) in aGVHD and 67% (2/3) in cGVHD. Of the 14 patients, adverse effects were observed in 9 patients (64.3%), including cytopenia, infection, and elevated alanine aminotransferase. In addition, seven reports on the treatment of SR-GVHD in children with ruxolitinib were included for systematic analysis, with the ORR ranging from 45 to 87% in aGVHD and 70–91% in cGVHD.

Conclusion

Given its effectiveness and safety, ruxolitinib could be used to treat SR-GVHD in children after HSCT.
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Metadata
Title
Ruxolitinib Treatment of Steroid-Refractory Graft-versus-Host Disease in Children: A Case Series and Review of the Literature
Authors
Wei-Ling Yan
Fen-Ying Zhao
Min-Er Gu
Nan Liu
Xiao-Ping Guo
Xiao-Jun Xu
Publication date
07-06-2023
Publisher
Springer International Publishing
Published in
Pediatric Drugs / Issue 5/2023
Print ISSN: 1174-5878
Electronic ISSN: 1179-2019
DOI
https://doi.org/10.1007/s40272-023-00577-8

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