白质脑病
医学
内科学
胼胝体
危险系数
队列
回顾性队列研究
病历
疾病
儿科
胃肠病学
肿瘤科
病理
置信区间
作者
Jarosław Dulski,Michael G Heckman,Julia M Nowak,Zbigniew K Wszolek
摘要
There is an unmet need for the treatment of colony-stimulating factor-1 receptor (CSF1R)-related leukoencephalopathy.To evaluate the association of glucocorticoids (GCs) with disease onset and progression in CSF1R variant carriers.Retrospective cohort study on CSF1R variants carriers (n = 41) whose medical records were collected at Mayo Clinic Florida from 2003 to 2023. We retrieved information on sex, ethnicity, family history, medications, disease onset, course and duration, neuroimaging features, and activities of daily living (ADL).Risk of developing symptoms was significantly lower for individuals who used GCs (n = 8) compared to individuals who did not (n = 33) (12.5% vs. 81.8%, hazard ratio [HR] = 0.10, P = 0.036). The risk of becoming dependent in ADL was markedly lower for the GCs group (0.0% vs. 43.8%, P = 0.006). White matter lesions and corpus callosum involvement were less common in the GCs group (62.5% vs. 96.6%, P = 0.026; 37.5% vs. 84.6%, P = 0.017; respectively).We found a protective association of GCs in CSF1R variant carriers against developing CSF1R-related leukoencephalopathy. We call for further studies to validate our findings and investigate the potential application of GCs in CSF1R-related leukoencephalopathy. © 2023 International Parkinson and Movement Disorder Society.
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