预防性应用芦可替尼减低儿童地中海贫血非亲缘和单倍体相合造血干细胞移植后GVHD的临床观察  

The Clinical Observation with Ruxolitinib as Graft-Versus-Host Disease Prophylaxis for Children with Thalassemia after Unrelated or Haploidentical Allo-Hematopoietic Stem Cell Transplantation

作　　者：陈亚玫[1] 洪秀理[1] 林进宗 石洁 鹿全意[1] CHEN Ya-Mei;HONG Xiu-Li;LIN Jin-Zong;SHI Jie;LU Quan-Yi(Department of Hematology,Zhongshan Hospital of Xiamen University,Xiamen 361004,Fujian Province,China)

机构地区：[1]厦门大学附属中山医院血液科,福建厦门361004

出　　处：《中国实验血液学杂志》2022年第5期1586-1589,共4页Journal of Experimental Hematology

基　　金：福建省科技计划引导项目(2019D009);厦门市科技计划指导项目(3502Z20209024)。

摘　　要：目的:评价芦可替尼预防儿童干细胞移植后移植物抗宿主病(GVHD)的有效性和安全性。方法:收集2020年3月-2021年3月期间本院完成的22例非亲缘供者和单倍体相合同胞供者造血干细胞移植的重型地中海贫血患儿,移植后+30到+100 d口服芦可替尼5 mg/d预防GVHD,观察治疗后患者急性GVHD、慢性GVHD,CMV、EBV病毒感染状况。结果:22例患者全部植入成功,5例患者诊断为急性GVHD,3例为II度以下皮肤GVHD,2例为II度肠道GVHD例,经治疗后痊愈,无死亡病例。全部患者随访时间超过21周,累计慢性GVHD 4例,其中局限性肝脏GVHD 3例,肺部GVHD 1例,经积极治疗后获得缓解。8例患者出现EBV copies升高(>3×10^(3)/ml),3例患者出现CMV copies数增加,给予减停免疫抑制剂、抗病毒治疗后恢复。没有患者发生CMV感染性疾病和EBV阳性的淋巴组织增殖异常,无移植相关的死亡。结论:对于替代供者移植的儿童患者,预防性应用芦可替尼可有效地减少GVHD的发生和严重程度,不影响造血恢复,改善了地贫儿童的移植后生存状况。Objective:To retrospectively analyze the efficacy and safety of ruxolitinib therapy for children with thalassemia after unrelated or haploidentical stem cell transplantation. Methods:From March2020to March2021, 22patients received successfully allogeneic hematopoietic stem cell transplantation in the Zhongshan Hospital of Xiamen University,from+ 30to100days,those patients received ruxolitinib therapy(2.5mg,twice daily)and all adverse reactions were observed,include aGVHD,cGVHD,CMV and EBV infection. Results: 22patients underwent allogeneic stem cell transplantation, 5patients were diagnosed as aGVHD, 3patients had grade I-II skin GVHD and2patients had grade II intestinal GVHD,those patients were cured. All patients were followed up for more than21weeks,4cases developed cGVHD,including3cases of localized liver GVHD and1case of pulmonary GVHD,those were relieved after active treatment.8patients had elevated EBV copies( > 3 × 10^(3)/ml),and 3 patients had increased CMV copies,the patients recovered after immunosuppressant and antiviral treatment. There was no CMV infection and EBV related post-transplantant lymphoproliferative disorders(PTLD),and no transplant related deaths. Conclusion:Ruxolitinib can effectively reduce the incidence and severity of GVHD without affecting the hematopoietic recovery,and improve the survival status of thalassemia children after transplantation.

关 键 词：地中海贫血 异基因造血干细胞移植 芦可替尼 移植物抗宿主病 儿童 

分 类 号：R556[医药卫生—血液循环系统疾病] R457.7[医药卫生—内科学]