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Abstract：
BACKGROUND: With the advent of innovative therapies including biologics and Janus kinase inhibitors, children with rheumatic diseases are more likely to have improved outcomes. Despite these advances, some children do not respond, or they, or their parents fear adverse events and seek other alternatives. Increasingly, private companies are offering mesenchymal stem cells (MSC) as an alternative, which are described as natural therapies for rheumatic diseases, often insinuating them as a cure. MSC have immunomodulatory properties, and transplantation of these stem cells have been used to successfully treat immunologic conditions like graft-versus-host disease. Lately, MSC research in adult lupus has been encouraging, but the clinical trials are still underway and in most, MSC therapy is not a standalone treatment. This retrospective case series will highlight three cases of pediatric refractory autoimmune disease whose parents sought out and received MSC therapy as a self-decision without first seeking medical advice from our specialty. The three families felt that their children were improved and in two believed that their child was cured. MSC have the potential of beneficial immunomodulation and may be a powerful tool in the therapy of rheumatic disease, but well controlled clinical trials are necessary and should be designed and monitored by experts in childhood rheumatic disease.
METHODS: Three children with three different rheumatic diseases; systemic lupus erythematosus, mixed connective tissue disease and juvenile idiopathic arthritis were under the care of pediatric rheumatology at a large, tertiary-care, teaching institution. Multiple non-biologic and biologic disease-modifying anti-rheumatic drugs failed to significantly decrease disease activity, and as a result, the families chose to undergo MSC therapy. After transplantation, all children improved per patient and parent report and tapered off conventional immunosuppressive drugs. No serious adverse events occurred in these three patients.
CONCLUSIONS: The three cases presented in this report reflect comparable beneficial outcomes and minimal risks published in adult studies. These were not controlled studies, however, and benefit was reported rather than documented. These cases suggest that MSC transplantation may prove a promising adjunctive treatment option; however, further research, development of standardized infusion therapy protocols, and well-designed monitored clinical trials are essential.
METHODS: Three children with three different rheumatic diseases; systemic lupus erythematosus, mixed connective tissue disease and juvenile idiopathic arthritis were under the care of pediatric rheumatology at a large, tertiary-care, teaching institution. Multiple non-biologic and biologic disease-modifying anti-rheumatic drugs failed to significantly decrease disease activity, and as a result, the families chose to undergo MSC therapy. After transplantation, all children improved per patient and parent report and tapered off conventional immunosuppressive drugs. No serious adverse events occurred in these three patients.
CONCLUSIONS: The three cases presented in this report reflect comparable beneficial outcomes and minimal risks published in adult studies. These were not controlled studies, however, and benefit was reported rather than documented. These cases suggest that MSC transplantation may prove a promising adjunctive treatment option; however, further research, development of standardized infusion therapy protocols, and well-designed monitored clinical trials are essential.
摘要：
背景：随着生物制剂和Janus激酶抑制剂等创新疗法的出现，患有风湿性疾病的儿童更有可能改善预后.尽管取得了这些进展,有些孩子没有回应，或者他们，或者他们的父母担心不良事件并寻求其他替代方案。越来越多,私营公司正在提供间充质干细胞(MSC)作为替代方案，被描述为风湿性疾病的自然疗法，经常暗示他们是一种治疗方法。MSC具有免疫调节特性，这些干细胞的移植已被成功地用于治疗免疫学疾病，如移植物抗宿主病。最近,MSC在成人狼疮中的研究一直令人鼓舞，但是临床试验仍在进行中，在大多数情况下，MSC治疗不是独立治疗。此回顾性病例系列将重点介绍三例小儿难治性自身免疫性疾病，其父母寻求并接受MSC治疗作为自我决定，而无需先寻求我们专业的医疗建议。三个家庭认为他们的孩子得到了改善，两个家庭认为他们的孩子已经治愈。MSC具有有益免疫调节的潜力，可能是治疗风湿性疾病的有力工具，但良好的对照临床试验是必要的，应该由儿童风湿性疾病专家设计和监测。
方法：三个患有三种不同风湿性疾病的儿童；系统性红斑狼疮，混合性结缔组织病和幼年特发性关节炎在儿科风湿病的照顾下，三级护理，教学机构。多种非生物和生物疾病改善抗风湿药物未能显着降低疾病活动，结果，这些家庭选择接受MSC治疗.移植后,根据患者和家长报告,所有儿童均有所改善,并逐渐减少了常规免疫抑制药物.3例患者均未发生严重不良事件。
结论：本报告中提供的三个案例反映了成人研究中发表的相当的有益结果和最小的风险。这些不是对照研究，然而,和效益报告，而不是记录。这些病例表明，MSC移植可能是一种有希望的辅助治疗选择；然而，进一步研究,标准化输液治疗方案的发展，和精心设计的监测临床试验是必不可少的。
方法：三个患有三种不同风湿性疾病的儿童；系统性红斑狼疮，混合性结缔组织病和幼年特发性关节炎在儿科风湿病的照顾下，三级护理，教学机构。多种非生物和生物疾病改善抗风湿药物未能显着降低疾病活动，结果，这些家庭选择接受MSC治疗.移植后,根据患者和家长报告,所有儿童均有所改善,并逐渐减少了常规免疫抑制药物.3例患者均未发生严重不良事件。
结论：本报告中提供的三个案例反映了成人研究中发表的相当的有益结果和最小的风险。这些不是对照研究，然而,和效益报告，而不是记录。这些病例表明，MSC移植可能是一种有希望的辅助治疗选择；然而，进一步研究,标准化输液治疗方案的发展，和精心设计的监测临床试验是必不可少的。
