摘要
目的回顾性分析在儿童造血干细胞移植中,鲁索利替尼治疗激素难治性或者复发的移植物抗宿主病(SR-GVHD)的疗效。方法选取我院12例异基因造血干细胞移植术(HSCT)后的SR-GVHD患儿。体重≥25kg,鲁索利替尼5mg,口服,每天2次,体重<25kg的患者,鲁索利替尼2.5mg,口服,每天2次。12例患儿中SR-aGVHD(n=5,均为Ⅱ～Ⅳ度),SR-cGVHD(n=8,局限型～广泛型)。同时排除在鲁索利替尼开始前2周内使用其他新的免疫抑制剂的患儿。其中1例患者分别在aGVHD及cGVHD时2次应用鲁索利替尼。4周为1个疗程,治疗期间患儿不耐受或者病情加重,定义为治疗失败。结果5例SR-aGVHD应用鲁索利替尼总反应率为4/5,1例患儿因病情加重而导致治疗失败。在SR-cGVHD应用鲁索利替尼的(n=8)例次患者中7例均有不同程度的好转,包括6例患儿完全好转,CR为6/8。总反应率(ORR)为7/8,1例患儿治疗失败。在鲁索利替尼使用期间,患儿出现不同程度的全血细胞减少和巨细胞病毒(CMV)血症,SR-aGVHD组中,出现全血细胞减少为2例次、CMV血症1例次;在SR-cGVHD组,出现全血细胞减少和CMV血症各1例次。中位随访1.1(0.5～1.7)年,所有患儿均存活。结论鲁索利替尼为儿童SR-aGVHD和SR-cGVHD的治疗提供了一种新的、有效的方法,但本次研究例数较少,需要进一步前瞻性大样本的研究。
Objective This study is to analyze retrospectively the effect of ruxolitinib treatment on steroid-refractory graft-versus-host disease（SR-GVHD） in pediatric hematopoietic stem cell（ HSCT）transplant patients. Methods 12 children with SR-GVHD were treated with ruxolitinib. Ruxolitinib was administered orally at 5 mg twice daily for children ≥ 25 kg or 2. 5 mg twice daily if 〈 25 kg. In this retrospective survey, Patients were classified as having SR-aGVHD（n = 5, all grade III or IV） or SRcGVHD（ n = 8, limited or diffused type）. One patient had used ruxolitinib in SR-aGVHD and SRcGVHD. We excluded patients who received new immune suppressive agents within 2 weeks before initiation of ruxolitinib from response analysis. Patients were defined as treatment failure if ruxolitinib was stopped before completion of 4 weeks of therapy because of adverse effects and progression of acute GVHD.Results The overall response rate（ORR） was 80 %（4/5） in SR-aGVHD, one patient was treatment failure, because the patient’s condition worsened. While for SR-cGVHD the ORR was 88%（7/8）including CR 75%（6/8） and PR 13%（1/8）, one patient was treatment failure, respectively. Cytopenia and CMV-reactivation were observed during ruxolitinib treatment in both SR-aGVHD（2/5, 40% and 1/5,20%） and SR-cGVHD（1/8, 13% and 1/8, 13%） patients. All patients are alive with a median followed up by 1. 1 years（0.5 ～ 1.7）. Conclusions Ruxolitinib may constitute a promising new treatment option for children of SR-aGVHD and SR-cGVHD that should be verified in a prospective trial and more clinical practice.
作者
岳燕
师晓东
YUE Yan;SHI Xiaodong(Department of Hematology,Capital Institute of Pediatrics,Beijing 100020,China)
出处
《中国小儿血液与肿瘤杂志》
CAS
2018年第4期211-216,224,共7页
Journal of China Pediatric Blood and Cancer
