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The Effectiveness of Ruxolitinib for Acute/Chronic Graft-versus-Host Disease in Children: A Retrospective Study
Drug Design, Development and Therapy ( IF 4.8 ) Pub Date : 2021-02-22 , DOI: 10.2147/dddt.s287218 Wenli Yang 1 , Guanghua Zhu 2 , Maoquan Qin 2 , Zhigang Li 3 , Bin Wang 2 , Jun Yang 2 , Tianyou Wang 2
Drug Design, Development and Therapy ( IF 4.8 ) Pub Date : 2021-02-22 , DOI: 10.2147/dddt.s287218 Wenli Yang 1 , Guanghua Zhu 2 , Maoquan Qin 2 , Zhigang Li 3 , Bin Wang 2 , Jun Yang 2 , Tianyou Wang 2
Affiliation
Objective: This study aimed to evaluate the effectiveness of Ruxolitinib for acute/chronic graft-versus-host disease in children.
Methods: This study was a retrospective trial. We analyzed the clinical characteristics of children who responded poorly to previous treatment for graft-versus-host disease (GVHD) and received ruxolitinib treatment after allogeneic hematopoietic stem cell transplantation (allo-HSCT) as an additional or replacement therapy.
Results: A total of 53 patients were analyzed: aGVHD and cGVHD. The overall response rate (ORR) to ruxolitinib was 75.5%. The ORR was 64.7% (11/17) in the aGVHD group including 6, 5, and 6 patients with partial responses (PRs), complete responses (CRs), and treatment failure, respectively. The ORR was 80.6% (29/36) in the cGVHD group including 10 with CRs and 19 with PRs. Five and 2 patients showed no response and treatment failure, respectively. Four and 14 patients were GVHD recurrence in aGVHD and cGVHD respectively. A total of 14 patients (39%) discontinued steroids and 8 patients (22.2%) reduced steroids. The incidence of obvious adverse events was 94.1% (16/17) in the aGVHD group, which was higher than that in the cGVHD group. Meanwhile, the prognosis of children with cGVHD was superior to that of children with aGVHD after treatment with ruxolitinib. During the ruxolitinib treatment, only 1 patient suffered a relapse of the primary tumor. Eleven patients also suffered transplantation-associated thrombotic microangiopathy (TA-TMA) after allo-HSCT.
Conclusion: Pediatric patients with GVHD (especially cGVHD) responded well to ruxolitinib treatment. Ruxolitinib can also be used as an alternative treatment for patients with TMA.
Keywords: ruxolitinib, graft-versus-host disease, allogeneic hematopoietic stem cell transplantation, Janus kinase1/2 inhibitor, pediatric patients
中文翻译：
鲁索替尼治疗儿童急性/慢性移植物抗宿主病的有效性：一项回顾性研究
目的：本研究旨在评估鲁索替尼治疗儿童急性/慢性移植物抗宿主病的有效性。
方法：本研究为回顾性试验。我们分析了对先前的移植物抗宿主病 (GVHD) 治疗反应不佳并在异基因造血干细胞移植 (allo-HSCT) 作为附加或替代疗法后接受鲁索替尼治疗的儿童的临床特征。
结果：共分析了 53 名患者：aGVHD 和 cGVHD。对鲁索替尼的总体缓解率 (ORR) 为 75.5%。aGVHD 组的 ORR 为 64.7% (11/17)，包括分别有 6、5 和 6 名部分缓解 (PR)、完全缓解 (CR) 和治疗失败的患者。cGVHD 组的 ORR 为 80.6% (29/36)，包括 10 例 CR 和 19 例 PR。5 名和 2 名患者分别显示无反应和治疗失败。在 aGVHD 和 cGVHD 中分别有 4 名和 14 名患者出现 GVHD 复发。共有 14 名患者 (39%) 停用类固醇，8 名患者 (22.2%) 减少类固醇。aGVHD组明显不良事件发生率为94.1%（16/17），高于cGVHD组。同时，鲁索替尼治疗后cGVHD患儿的预后优于aGVHD患儿。在鲁索替尼治疗期间，仅有1名患者原发肿瘤复发。11 名患者在 allo-HSCT 后还患有移植相关血栓性微血管病 (TA-TMA)。
结论：患有 GVHD（尤其是 cGVHD）的儿科患者对鲁索替尼治疗反应良好。鲁索替尼也可用作 TMA 患者的替代治疗。
关键词：鲁索替尼，移植物抗宿主病，异基因造血干细胞移植，Janus 激酶1/2 抑制剂，儿科患者
更新日期：2021-04-20
Methods: This study was a retrospective trial. We analyzed the clinical characteristics of children who responded poorly to previous treatment for graft-versus-host disease (GVHD) and received ruxolitinib treatment after allogeneic hematopoietic stem cell transplantation (allo-HSCT) as an additional or replacement therapy.
Results: A total of 53 patients were analyzed: aGVHD and cGVHD. The overall response rate (ORR) to ruxolitinib was 75.5%. The ORR was 64.7% (11/17) in the aGVHD group including 6, 5, and 6 patients with partial responses (PRs), complete responses (CRs), and treatment failure, respectively. The ORR was 80.6% (29/36) in the cGVHD group including 10 with CRs and 19 with PRs. Five and 2 patients showed no response and treatment failure, respectively. Four and 14 patients were GVHD recurrence in aGVHD and cGVHD respectively. A total of 14 patients (39%) discontinued steroids and 8 patients (22.2%) reduced steroids. The incidence of obvious adverse events was 94.1% (16/17) in the aGVHD group, which was higher than that in the cGVHD group. Meanwhile, the prognosis of children with cGVHD was superior to that of children with aGVHD after treatment with ruxolitinib. During the ruxolitinib treatment, only 1 patient suffered a relapse of the primary tumor. Eleven patients also suffered transplantation-associated thrombotic microangiopathy (TA-TMA) after allo-HSCT.
Conclusion: Pediatric patients with GVHD (especially cGVHD) responded well to ruxolitinib treatment. Ruxolitinib can also be used as an alternative treatment for patients with TMA.
Keywords: ruxolitinib, graft-versus-host disease, allogeneic hematopoietic stem cell transplantation, Janus kinase1/2 inhibitor, pediatric patients
中文翻译：
鲁索替尼治疗儿童急性/慢性移植物抗宿主病的有效性：一项回顾性研究
目的：本研究旨在评估鲁索替尼治疗儿童急性/慢性移植物抗宿主病的有效性。
方法：本研究为回顾性试验。我们分析了对先前的移植物抗宿主病 (GVHD) 治疗反应不佳并在异基因造血干细胞移植 (allo-HSCT) 作为附加或替代疗法后接受鲁索替尼治疗的儿童的临床特征。
结果：共分析了 53 名患者：aGVHD 和 cGVHD。对鲁索替尼的总体缓解率 (ORR) 为 75.5%。aGVHD 组的 ORR 为 64.7% (11/17)，包括分别有 6、5 和 6 名部分缓解 (PR)、完全缓解 (CR) 和治疗失败的患者。cGVHD 组的 ORR 为 80.6% (29/36)，包括 10 例 CR 和 19 例 PR。5 名和 2 名患者分别显示无反应和治疗失败。在 aGVHD 和 cGVHD 中分别有 4 名和 14 名患者出现 GVHD 复发。共有 14 名患者 (39%) 停用类固醇，8 名患者 (22.2%) 减少类固醇。aGVHD组明显不良事件发生率为94.1%（16/17），高于cGVHD组。同时，鲁索替尼治疗后cGVHD患儿的预后优于aGVHD患儿。在鲁索替尼治疗期间，仅有1名患者原发肿瘤复发。11 名患者在 allo-HSCT 后还患有移植相关血栓性微血管病 (TA-TMA)。
结论：患有 GVHD（尤其是 cGVHD）的儿科患者对鲁索替尼治疗反应良好。鲁索替尼也可用作 TMA 患者的替代治疗。
关键词：鲁索替尼，移植物抗宿主病，异基因造血干细胞移植，Janus 激酶1/2 抑制剂，儿科患者
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