【摘要】 目的通过观察大剂量化疗(HDC)并自体外周血干细胞移植(APBSCT)治疗重症原发性干燥综合征(pSS)患者治疗前后 T 细胞亚群和 T 细胞受体(TCR)克隆谱型的变化,探讨移植的免疫重建过程。方法 2例长期糖皮质激素和免疫抑制剂治疗病情不能缓解的重症 pSS 患者,给予HDC 及 APBSCT 治疗。流式细胞仪检测 T 细胞亚群,观察治疗前后 T 细胞亚群的变化。RT-PCR 扩增患者外周血 TCRV 区24个β亚家族序列,阳性的亚家族行荧光标记的二次 PCR,用分析 TCRVβ亚家族 CDR3长度的方法检测 T 细胞克隆性,以及移植前后 T 细胞克隆谱的变化。结果 2例重症 pSS患者顺利完成 APBSCT 全过程,T 细胞亚群有不同程度的恢复,移植后 TCRVβ有由寡克隆向多克隆转化的趋势。结论本组2例重症 pSS 患者 HDC 并 APBSCT 治疗后病情得以控制,免疫功能在3个月时已有恢复的趋势,但其可行性与安全性有待大样本及随诊证实。更多还原

【Abstract】 Objectives To assess the safety and efficacy of high-dose immunosuppression and autologous peripheral blood cell transplantation(APBSCT)in severe and refractory primary Sjgren’s syndrome(pSS)and to analyze immune reconstitution in pSS.Methods Two patients with severe and refractory primary pSS were included in this study.They suffered still with active pSS despite the use of prednisone and immunosupression agents.A regimen of high-dose immunosuppression and APBSCT was carried out for them.Dynamic T cell subgroup was tested with flow cytometry before and after PBSCT and the diversity of T cell receptor repertoire and CDR3 spectrum with RT-PCR and genescan.Results Both of the pSS cases underwent PBSCT smoothly.Clinical assessments showed improvement.Immune reconstruction lagged behind hematopoietic reconstitution.The skew of T cell receptor repertoire was somewhat corrected and CDR3 spectrum changed from oligoclonality to poly-clonality.Conclusion High dose chemotyherapy(HDC) and APBSCT are feasible and safe and can result in short-term or middle-term improvement of disease in patients with severe pSS which is refractory to conventional treatment.It is observed in this study that immune reconstruction recovered 3 moths after the treatment.Long-term efficacy of HDC+PBSCT in pSS should be studied in large number of cases with follow up of longer time.更多还原