[Clinical Efficacy of Haplo-HSCT of ATG Combined with PTCy for Children with Myelodysplastic Syndrome].

• DOI: 10.19746/j.cnki.issn.1009-2137.2022.02.032
• Publication Date: 2022-04-01
• Authors: Zeliang Song; Rong Liu; Tao Hu; Junhui Li; Chaoxia Zhang; Lei Zhang; Dixiao Zhong; Mei Yue; Xiaodong Shi
• Keywords: Cytopenia; Median follow-up

To investigate the efficacy and safety of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) in combination ATG post-transplant cyclophosphamide (PTCy) -induced immune tolerance after treatment childhood myelodysplastic syndromes(MDS）.From July 2016 to November 2020, a total 8 children with MDS receiving allo-HSCT combined PTCy-induced our hospital were enrolled, whose clinical data retrospected analyzed.Median age at diagnosis (1 male 7 females) was 6.4 (range, 10 months 15 years) years old. The median medical history 2.7 3 years). Among patients, cases diagnosed refractory cytopenia one anemia excess blasts. HSC donors father, mother or brother patients HLA matching 6-9/12 loci identical. All healthy didn't carry same pathogenic genes as recipients. 36.4 25 49) mononuclear (MNC) number graft 19.8, ranging (13.2-47.3)×108/kg, CD34+ 11.8×106/kg, (5.0-18.3)×106/kg. Graft-versus-host disease prophylactic regimen started on day 4 transplantation, which (50 mg/kg·d) administered by intravenous infusion. From 5 low-dose tacrolimus infusion mycophenolate mofetil orally. time neutrophil platelet engraftment 12.6 (rang, 11 15) days 13.3 18) days, respectively. achieved full donor chimerism transplantation. follow-up 1 032 747 536) days. Both overall survival rate disease-free 100%.Haplo-HSCT is safe effective for MDS.ATG联合PTCy的Haplo-HSCT方案治疗儿童骨髓增生异常综合征.探讨ATG联合后置环磷酰胺（PTCy）诱导免疫耐受的单倍体型造血干细胞移植（haplo-HSCT）方案治疗儿童骨髓增生异常综合征（MDS）的疗效及安全性.回顾并分析2016年7月至2020年11月在本中心接受ATG联合PTCy诱导免疫耐受的Haplo-HSCT方案治疗的8例儿童MDS患者的临床资料.8例儿童MDS患者中，男1例，女7例，中位年龄6.4岁（10个月-15岁），病史中位数27年（3个月-8年），7例诊断为难治性血细胞减少症，1例诊断为难治性贫血伴原始细胞增多。供者为患者父亲、母亲或哥哥，HLA配型（6-9）/12位点相合，供者经筛查均为健康供者，未携带受者致病基因，中位年龄364（25-49）岁，回输单个核细胞中位数 19.8(13.2-47.3) ×108/kg，CD34+细胞中位数11.8（5.0-18.3）×106/kg。移植后3至4 d予环磷酰胺（Cy/CTX）50 mg/(kg·d)静脉滴注，移植后5 d起予小剂量他克莫司（FK506）静脉滴注、吗替麦考酚酯（MMF）口服预防移植物抗宿主病。粒细胞和血小板植入时间分别为12.6（11-15）和13.3（11-18）d，所有患者在植入时均获得完全的供体细胞嵌合。中位随访时间为1032（747-1536）d，总体生存率及无病生存率均为 100.ATG联合PTCy诱导免疫耐受的Haplo-HSCT方案是治疗儿童MDS安全有效的方法.