造血干细胞移植治疗多发性硬化症和其他神经退行性疾病的全球在线研究

Q1 Medicine
Peter K. Panegyres, Jodi Russell, Huei-Yang Chen, Mariella Panegyres
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引用次数: 0

摘要

本研究的目的是了解造血干细胞移植(HSCT)在多发性硬化症(MS)等神经免疫疾病中的摄取情况。方法一个独立的大学附属研究机构对接受过HSCT的人进行了一项全球在线调查,检查人口统计学、治疗方案和有效性。结果在271名参与者中,223名获得了有用的数据;35-54岁的女性占73.5%。大多数人的家庭收入超过5万美元,大多数参与者来自澳大利亚和美国。近94.6%的人患有多发性硬化症,大多数人在俄罗斯接受治疗(38.7%),78.1%的人接受非清髓性移植。近一半的参与者花费在5万美元到74,999美元之间。54.5%的神经科医生不支持患者接受造血干细胞移植。大约85.5%的参与者认为HSCT帮助他们在移植后数周到数年内控制疾病,9.5%的参与者推荐治疗。移植后扩展残疾状态评分平均降低1.2 (95% CI: 0.97-1.41;n = 197;p < 0.01;T: 10.7, df: 196)。结论尽管成本高昂,但参与者仍支持HSCT,并将其推荐给他人。数据显示在减少多发性硬化症的残疾方面有一定的益处,并为大型随机对照试验提供了理由。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

A global online study of haematopoietic stem cell transplantation in multiple sclerosis and other neurodegenerative disorders

A global online study of haematopoietic stem cell transplantation in multiple sclerosis and other neurodegenerative disorders

Background

The objective of this study was to understand the uptake of hemopoietic stem cell transplantation (HSCT) in neuroimmunological disorders like multiple sclerosis (MS).

Method

An independent University affiliated research organization conducted a global online survey of people having had HSCT, examining demographics, treatment protocol, and effectiveness.

Results

Of 271 participants, useful data were available in 223; women aged 35–54 accounted for 73.5%. Most had a household income greater than US$50,000, and the majority of participants were from Australia and the United States. Nearly 94.6% of people suffer from MS. Most had their treatment in Russia (38.7%) and 78.1% had nonmyeloablative transplants. Nearly half of the participants spent between US$50,000 to US$74,999. There were 54.5% of neurologists who did not support their patients having HSCT. Around 85.5% of participants believed HSCT helped them manage their disease from weeks to years after transplantation, and treatment was recommended by 9.5% of participants. The average reduction in Expanded Disability Status Score after transplantation was 1.2 (95% CI: 0.97–1.41; N = 197; p < 0.01; t: 10.7, df: 196).

Conclusion

Participants were supportive of HSCT despite the costs and would recommend it to others. The data suggest some benefit in minimizing disability in MS and provides justification for large randomized controlled trials.

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来源期刊
CiteScore
6.70
自引率
0.00%
发文量
195
审稿时长
35 weeks
期刊介绍: This journal aims to promote progress from basic research to clinical practice and to provide a forum for communication among basic, translational, and clinical research practitioners and physicians from all relevant disciplines. Chronic diseases such as cardiovascular diseases, cancer, diabetes, stroke, chronic respiratory diseases (such as asthma and COPD), chronic kidney diseases, and related translational research. Topics of interest for Chronic Diseases and Translational Medicine include Research and commentary on models of chronic diseases with significant implications for disease diagnosis and treatment Investigative studies of human biology with an emphasis on disease Perspectives and reviews on research topics that discuss the implications of findings from the viewpoints of basic science and clinical practic.
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