Division of Paediatric Rheumatology, Capital Institute of Pediatrics, No. 2 Yabao Road, Chaoyang District, Beijing, 100020, China.
Clin Rheumatol. 2013 Dec;32(12):1727-34. doi: 10.1007/s10067-013-2324-1. Epub 2013 Aug 8.
This study attempts to evaluate the outcome of autologous peripheral blood hematopoietic stem cell transplantation (auto-PBHSCT) in patients with severe paediatric systemic lupus erythematosus (SLE). Five patients (n = 2 females, n = 3 males) with severe or refractory paediatric SLE received autologous peripheral blood CD34+ cell transplants between July 2005 and February 2009. The patients ranged in age from 6 to 14 years, and the course of disease extended over a period from 5 to 90 months. All of the patients received conventional therapy for 3 to 87 months. After their discharge from the hospital, the patients continued to maintain their regular follow-up visits and basic quality of life. The patients exhibited decreased immune function after the auto-PBHSCT. The CD4+ and CD19+ cells were significantly reduced. Viremia occurred in four patients 2 months after the transplantation. All of the patients went into clinical remission in 3-6 months. The severity of encephalopathy, nephritis and organ damage declined in varying degrees. The disease recurred in patient 2 at 9 months and in patient 4 at 12 months after the transplantation. Because the disease was relatively mild, we were able to administer small doses of glucocorticoids that were sufficient to control the course of the disease. Macrophage activation syndrome occurred in patient 3 at 18 months after the transplantation. At the end of the follow-up period, three of the five patients were completely off their medications. Another two patients sustained small doses of glucocorticoids. The developmental levels of these patients were comparable to those of normal children at the end of the follow-up. The quality of life improved significantly. The auto-PBHSCT is effective for severe and refractory paediatric SLE. The incidence of lethal infection and other adverse reactions is low. Long-term remission can be achieved. A milder form of the disease may have recurred after the transplantation.
本研究旨在评估自体外周血造血干细胞移植(auto-PBHSCT)治疗儿童严重系统性红斑狼疮(SLE)的疗效。2005 年 7 月至 2009 年 2 月，5 例严重或难治性儿童 SLE 患者接受了自体外周血 CD34+细胞移植。患者年龄 614 岁，病程 590 个月。所有患者均接受常规治疗 387 个月。出院后，患者继续定期随访，保持基本生活质量。自体 PBHSCT 后患者免疫功能下降，CD4+、CD19+细胞明显减少，4 例患者移植后 2 个月发生病毒血症。所有患者均在 36 个月内临床缓解，神经精神性狼疮、肾炎及器官损害程度不同程度减轻。2 例患者在移植后 9 个月、4 例患者在移植后 12 个月复发，病情均较轻，给予小剂量糖皮质激素即可控制病情。3 例患者在移植后 18 个月发生巨噬细胞活化综合征。随访结束时，5 例患者中有 3 例完全停药，2 例维持小剂量糖皮质激素。随访结束时，所有患者的发育水平与正常儿童相当，生活质量明显改善。自体 PBHSCT 治疗儿童严重、难治性 SLE 有效，感染等致死性不良反应发生率低，可长期缓解，移植后病情较轻者可能复发。
