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.

中文翻译：

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鲁索替尼治疗儿童类固醇难治性移植物抗宿主病：病例系列和文献回顾

背景

鲁索替尼已越来越多地用于治疗异基因造血干细胞移植（allo-HSCT）患者的类固醇难治性移植物抗宿主病（SR-GVHD）。然而，有关鲁索替尼在儿童中使用的数据有限。

客观的

本研究旨在评估鲁索替尼治疗儿童 SR-GVHD 的疗效和毒性。

患者和方法

回顾性分析2018年6月至2020年12月我中心allo-HSCT后出现SR-GVHD并接受鲁索替尼治疗的患者资料。收集患者特征、鲁索替尼剂量、反应、毒性和生存数据。

结果

共有 14 名儿童患者在 allo-HSCT 后被诊断为 SR-GVHD，并接受了鲁索替尼治疗。患者年龄从3个月至12岁不等。鲁索替尼的剂量范围为2.5毫克每天两次至7.5毫克每天两次，主要根据患者体重而定。总缓解率 (ORR) 为 64.3% (9/14)，其中 aGVHD 为 63.6% (7/11)，cGVHD 为 67% (2/3)。14 名患者中，9 名患者（64.3%）出现不良反应，包括血细胞减少、感染和丙氨酸氨基转移酶升高。此外，还纳入了7篇关于鲁索替尼治疗儿童SR-GVHD的报告进行系统分析，aGVHD的ORR范围为45%至87%，cGVHD的ORR范围为70%至91%。

结论

鉴于其有效性和安全性，鲁索替尼可用于治疗 HSCT 后儿童的 SR-GVHD。