CD34^+细胞自身移植治疗儿童难治型系统性红斑狼疮

Primary report on the application of CD34^+ autologous peripheral progenitor cell transplant in the treatment of children with refractory SLE

目的探讨治疗儿童难治型系统性红斑狼疮 (SLE)积极、有效的方法。方法对 2例病程分别为 5年和 7年 ,狼疮肾Ⅲ级和Ⅳ级 ,主要表现为持续性血小板减少、蛋白尿和浆膜炎的患儿进行CD34+ 细胞分选的自身干细胞移植。首先经惠尔血动员、CS 30 0 0血细胞分离机获取单个核细胞 ,通过CliniMACSCD34+ 细胞分选仪分别得到了 1.7× 10 6/kg及 1.0× 10 6/kgCD34+细胞 ,采集物中分别尚存 2× 10 5/kg和 1× 10 4/kg的CD3+ 细胞。用CTX 5 0mg/kg·d× 4d +ATG(FresenniusS 5mg/kg·d× 3d)预处理。结果两患者分别于 +9d和 +7d获粒细胞重建 ,自 +15d起血小板维持于正常水平。现已分别随访 13月和 6月 ,原发病症状完全消失 ,自身免疫相关抗体全部转阴 ,但细胞免疫功能仍未恢复 ,CD4细胞仍处于低水平。结论CD34+ 细胞分选的自身干细胞移植治疗儿童难治型红斑狼疮近期疗效满意。

Objective To explore the active and effective way to treat children with refractory systemic lupus erythematosus (SLE). Methods Two children with 5-year and 7-year duration of the lupus nephropathy at III and IV grade,respectively, mainly presented with persistent thrombocytopenia, proteinuria, and serositis received CD34 + autologous peripheral progenitor cells transplantation. Mobilized with G-CSF and collected with CS-3000 cell separator, passed through the CliniMacs CD34 + cell selection device, the count of CD34 + cells reached 1.0×10 6 /kg and 1.7×10 6 /kg,respectively, with a remainder of 2.0×10 5/kg and 1.0×10 4 /kg of CD3 + cells individually. The conditioning regimen consisted of cyclophosphomide (50 mg/kg·d for 4 days) plus ATG (Fresennius S 5 mg/kg·d for 3 days). Results Neutrophils recovered on +9 and +7 days after transplantation respectively in these 2 cases. Beginning from +15 days, the platelet count also recovered. Up to now, these 2 children have been followed up for 13 months and 6 months individually with disappearance of all the original presentations and autoantibodies related to autoimmune disorders, but the cell-mediated immunity was not recovered yet with the CD4 + cell level still remaining in the lower level. Conclusion The effect of CD34 + autologous prepheral progenitor cell transplantation on children with refractory SLE was satisfied in short term.