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

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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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Authors and Affiliations

  1. Department of Hematology and Oncology, Children’s Hospital, Zhejiang University School of Medicine, No. 57 Zhugan Lane, Yan-an Road, Gongshu District, Hangzhou, Zhejiang, China

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

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Correspondence to Xiao-Jun Xu.

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Competing interests

Wei-Ling Yan, Fen-Ying Zhao, Min-Er Gu, Nan Liu, Xiao-Ping Guo, and Xiao-Jun Xu declare that they have no conflicts of interest that might be relevant to the content of this manuscript.

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No external funding was used in the preparation of this manuscript.

Ethics approval

This work has been approved by the ethic committee of Children’s Hospital, Zhejiang University School of Medicine, approval no. 2022-IRB-209.

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Written informed consent was obtained from patients’ guardians.

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Not applicable.

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Not applicable.

Author contributions

Xiao-Jun Xu contributed to the study conception and design. Material preparation and data collection were performed by Wei-Ling Yan, Min-Er Gu, Nan Liu, and Xiao-Ping Guo. Data interpretation and analyzing were performed by Xiao-Jun Xu and Fen-Ying Zhao. The first draft of the manuscript was written by Wei-Ling Yan. Xiao-Jun Xu commented on previous versions of the manuscript. All authors read and approved the final manuscript.

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Yan, WL., Zhao, FY., Gu, ME. et al. Ruxolitinib Treatment of Steroid-Refractory Graft-versus-Host Disease in Children: A Case Series and Review of the Literature. Pediatr Drugs 25, 577–584 (2023). https://doi.org/10.1007/s40272-023-00577-8

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  • DOI: https://doi.org/10.1007/s40272-023-00577-8

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