Panegyres Peter K, Russell Jodi, Chen Huei-Yang, Panegyres Mariella
Neurodegenerative Disorders Research Pty Ltd West Perth Western Australia Australia.
The University of Western Australia Nedlands Western Australia Australia.
Chronic Dis Transl Med. 2022 Aug 26;9(1):39-43. doi: 10.1002/cdt3.44. eCollection 2023 Mar.
The objective of this study was to understand the uptake of hemopoietic stem cell transplantation (HSCT) in neuroimmunological disorders like multiple sclerosis (MS).
An independent University affiliated research organization conducted a global online survey of people having had HSCT, examining demographics, treatment protocol, and effectiveness.
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; = 197; < 0.01; : 10.7, : 196).
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.
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应用情况。
一个独立的大学附属研究机构对接受过HSCT的人群进行了一项全球在线调查，调查内容包括人口统计学、治疗方案和疗效。
在271名参与者中，223人提供了有用数据；35 - 54岁的女性占73.5%。大多数人的家庭收入超过5万美元，且大多数参与者来自澳大利亚和美国。近94.6%的人患有MS。大多数人在俄罗斯接受治疗（38.7%），78.1%的人接受了非清髓性移植。近一半的参与者花费在5万至74,999美元之间。有54.5%的神经科医生不支持他们的患者接受HSCT。约85.5%的参与者认为HSCT在移植后的数周乃至数年帮助他们控制了病情，9.5%的参与者推荐了该治疗方法。移植后扩展残疾状态评分的平均降低值为1.2（95%置信区间：0.97 - 1.41；= 197；< 0.01；：10.7，：196）。
尽管费用高昂，参与者仍支持HSCT，并会向他人推荐。数据表明HSCT在减轻MS残疾方面有一定益处，为大型随机对照试验提供了依据。
