Irene Cortese, Gina Norato, Patrick R Harrington, Therri Usher, Ilaria Mainardi, Guillaume Martin-Blondel, Paola Cinque, Eugene O Major, Virginia Sheikh
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引用次数: 0
摘要
进行性多灶性白质脑病是一种罕见的破坏性脱髓鞘疾病，由 JC 病毒（JCV）引起，目前尚无治疗药物获批。为了在满足这一尚未满足的医疗需求方面取得进展，需要在临床试验设计方面进行创新。脑脊液中的定量 JCV DNA 有可能成为临床试验中进行性多灶性白质脑病疾病和治疗反应的重要生物标志物，从而加快疗法的开发，神经影像学和神经丝轻链等其他体液生物标志物也是如此。具体来说，CSF 中的 JCV DNA 可作为临床试验的入选标准、分层因素或临床结果预测因子。对进行性多灶性白质脑病候选生物标志物的研究可能会为其他罕见病的生物标志物开发提供参考。
Biomarkers for progressive multifocal leukoencephalopathy: emerging data for use of JC virus DNA copy number in clinical trials.
Progressive multifocal leukoencephalopathy is a rare but devastating demyelinating disease caused by the JC virus (JCV), for which no therapeutics are approved. To make progress towards addressing this unmet medical need, innovations in clinical trial design are needed. Quantitative JCV DNA in CSF has the potential to serve as a valuable biomarker of progressive multifocal leukoencephalopathy disease and treatment response in clinical trials to expedite therapeutic development, as do neuroimaging and other fluid biomarkers such as neurofilament light chain. Specifically, JCV DNA in CSF could be used in clinical trials as an entry criterion, stratification factor, or predictor of clinical outcomes. Insights from the investigation of candidate biomarkers for progressive multifocal leukoencephalopathy might inform approaches to biomarker development for other rare diseases.
期刊介绍：
The Lancet Neurology is the world-leading clinical neurology journal. It publishes original research that advocates for change in, or sheds light on, neurological clinical practice. The topics covered include cerebrovascular disease, Alzheimer's disease and other dementias, epilepsy, migraine, neurological infections, movement disorders, multiple sclerosis, neuromuscular disorders, peripheral nerve disorders, pediatric neurology, sleep disorders, and traumatic brain injury.
The journal publishes a range of article types, including Articles (including randomized clinical trials and meta-analyses), Review, Rapid Review, Comment, Correspondence, and Personal View. It also publishes Series and Commissions that aim to shape and drive positive change in clinical practice and health policy in areas of need in neurology.
The Lancet Neurology is an internationally trusted source of clinical, public health, and global health knowledge. It has an Impact Factor of 48.0, making it the top-ranked clinical neurology journal out of 212 journals worldwide.
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