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接受神经干细胞治疗脑瘫的意愿:对澳大利亚脑瘫社区的调查。

Acceptability of neural stem cell therapy for cerebral palsy: survey of the Australian cerebral palsy community.

机构信息

The Ritchie Centre, Hudson Institute of Medical Research, Clayton, VIC, Australia.

Department of Obstetrics and Gynaecology, Monash University, Clayton, VIC, Australia.

出版信息

Stem Cell Res Ther. 2023 Feb 3;14(1):18. doi: 10.1186/s13287-023-03246-2.

Abstract

BACKGROUND

Neural stem cells (NSCs) have the potential to engraft and replace damaged brain tissue, repairing the damaged neonatal brain that causes cerebral palsy (CP). There are procedures that could increase engraftment of NSCs and may be critical for efficacy, but hold notable risks. Before clinical trials progress, it is important to engage with the CP community to understand their opinions. The aim of this study was to determine the acceptability of NSC therapy for CP in the CP community.

METHODS

Australian residents with CP and parents/carers of those with CP completed a questionnaire to determine their willingness to use NSCs from three sources (fetal, embryonic and induced pluripotent stem cells) and their willingness to undergo accompanying procedures (neurosurgery, immunosuppression) that carry potential risks. To further explore their views, participants also answered free text questions about their ethical concerns regarding the source of NSCs and their perceptions of meaningful outcomes following NSC treatment.

RESULTS

In total, 232 responses were analyzed. Participants were willing to use NSCs from all three cell sources and were willing to undergo NSC therapy despite the need for neurosurgery and immunosuppression. Participants identified a range of outcome domains considered important following NSC treatment including gross motor function, quality of life, independence and cognitive function.

CONCLUSIONS

Hypothetical NSC therapy was acceptable to the Australian CP community. This study has identified important findings from the CP community which can be used to inform future NSC research, including the design of clinical trials which may help to increase recruitment, compliance and participant satisfaction.

摘要

背景

神经干细胞(NSCs)具有植入和替代受损脑组织的潜力,可以修复导致脑瘫(CP)的新生儿脑损伤。有一些程序可以增加 NSCs 的植入率,这对疗效可能至关重要,但也存在显著风险。在临床试验进展之前,与 CP 社区进行接触以了解他们的意见非常重要。本研究旨在确定 CP 社区对 NSC 治疗 CP 的接受程度。

方法

澳大利亚 CP 患者及其父母/照顾者完成了一份问卷,以确定他们对来自三种来源(胎儿、胚胎和诱导多能干细胞)的 NSCs 的使用意愿,以及他们对伴随治疗(神经外科手术、免疫抑制)的意愿,这些治疗存在潜在风险。为了进一步探讨他们的观点,参与者还回答了关于 NSCs 来源的伦理问题以及对 NSC 治疗后有意义的结果的看法的自由文本问题。

结果

共分析了 232 份回复。参与者愿意使用来自所有三种细胞来源的 NSCs,并且愿意接受 NSC 治疗,尽管需要神经外科手术和免疫抑制。参与者确定了一系列被认为重要的 NSC 治疗后结果领域,包括粗大运动功能、生活质量、独立性和认知功能。

结论

假设的 NSC 治疗对澳大利亚 CP 社区是可以接受的。本研究从 CP 社区中确定了重要的发现,这些发现可用于为未来的 NSC 研究提供信息,包括临床试验的设计,这可能有助于增加招募、依从性和参与者满意度。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9338/9898914/60b3374627d4/13287_2023_3246_Fig1_HTML.jpg

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