Department of Clinical Nutrition, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, People's Republic of China.
Department of Hematology and Oncology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing Key Laboratory of Pediatric Hematology Oncology, Key Laboratory of Major Diseases in Children, Ministry of Education, National Key Discipline of Pediatrics, Beijing, People's Republic of China.
Drug Des Devel Ther. 2021 Feb 22;15:743-752. doi: 10.2147/DDDT.S287218. eCollection 2021.
This study aimed to evaluate the effectiveness of Ruxolitinib for acute/chronic graft-versus-host disease in children.
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.
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.
Pediatric patients with GVHD (especially cGVHD) responded well to ruxolitinib treatment. Ruxolitinib can also be used as an alternative treatment for patients with TMA.
本研究旨在评估芦可替尼治疗儿童急性/慢性移植物抗宿主病（GVHD）的疗效。
本研究为回顾性试验。我们分析了既往治疗GVHD效果不佳且在异基因造血干细胞移植（allo-HSCT）后接受芦可替尼作为附加或替代治疗的患儿的临床特征。
共分析了 53 例患者：急性 GVHD 和慢性 GVHD。芦可替尼的总体缓解率（ORR）为 75.5%。急性 GVHD 组的 ORR 为 64.7%（11/17），包括 6、5 和 6 例部分缓解（PR）、完全缓解（CR）和治疗失败。慢性 GVHD 组的 ORR 为 80.6%（29/36），包括 10 例 CR 和 19 例 PR。5 例和 2 例患者无反应和治疗失败。4 例和 14 例患者在急性 GVHD 和慢性 GVHD 中分别出现 GVHD 复发。共有 14 例（39%）患者停用了类固醇，8 例（22.2%）患者减少了类固醇的用量。急性 GVHD 组不良反应发生率为 94.1%（17/18），高于慢性 GVHD 组。同时，芦可替尼治疗后慢性 GVHD 患儿的预后优于急性 GVHD 患儿。在芦可替尼治疗期间，仅 1 例患者发生原发性肿瘤复发。11 例患者在 allo-HSCT 后也发生了移植相关血栓性微血管病（TA-TMA）。
GVHD 患儿（尤其是慢性 GVHD 患儿）对芦可替尼治疗反应良好。芦可替尼也可作为 TMA 患者的替代治疗方法。
