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.
Paediatr Drugs. 2023 Sep;25(5):577-584. doi: 10.1007/s40272-023-00577-8. Epub 2023 Jun 7.
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.
This study aimed to assess the efficacy and toxicity of ruxolitinib in the treatment of SR-GVHD in children.
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.
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.
Given its effectiveness and safety, ruxolitinib could be used to treat SR-GVHD in children after HSCT.
鲁索利替尼已越来越多地用于治疗异基因造血干细胞移植（allo-HSCT）患者的激素难治性移植物抗宿主病（SR-GVHD）。然而，儿童使用鲁索利替尼的数据有限。
本研究旨在评估鲁索利替尼治疗儿童 SR-GVHD 的疗效和毒性。
回顾性分析了 2018 年 6 月至 2020 年 12 月期间在我院接受 allo-HSCT 后发生 SR-GVHD 并接受鲁索利替尼治疗的患者的数据。收集了患者的特征、鲁索利替尼的剂量、反应、毒性和生存数据。
共有 14 例儿童 allo-HSCT 后诊断为 SR-GVHD，并接受鲁索利替尼治疗。患者年龄 3 个月至 12 岁。鲁索利替尼的剂量范围为 2.5mg，每日两次至 7.5mg，每日两次，主要根据患者体重而定。总总体缓解率（ORR）为 64.3%（9/14），其中急性移植物抗宿主病（aGVHD）为 63.6%（7/11），慢性移植物抗宿主病（cGVHD）为 67%（2/3）。14 例患者中，9 例（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 篇关于儿童用鲁索利替尼治疗 SR-GVHD 的研究进行系统分析，aGVHD 的 ORR 为 45-87%，cGVHD 的 ORR 为 70-91%。
鉴于其有效性和安全性，鲁索利替尼可用于治疗 HSCT 后儿童的 SR-GVHD。
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