Association of Dilated Perivascular Spaces and Disease Severity in Patients With Huntington Disease.
Association of Dilated Perivascular Spaces and Disease Severity in Patients With Huntington Disease.
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DOI：
10.1212/wnl.0000000000011121
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发表时间：
2021-02-09
期刊：
影响因子：
9.9
通讯作者：
Rosas HD
中科院分区：
文献类型：
--
作者：
Chan ST;Mercaldo ND;Ravina B;Hersch SM;Rosas HD
量化早期亨廷顿病 (HD) 患者皮质下前脑扩张的血管周围空间 (PVS) 的体积百分比，并探讨 PVS 与疾病严重程度之间的关系。对马萨诸塞州综合医院的 25 名 HD 患者和 23 名年龄匹配的健康对照者进行了 MRI 扫描。使用新算法分析成像数据以确定局部 PVS 体积。使用分数逻辑回归分析来量化区域PVS体积百分比与（1）疾病指定（HD或对照）和（2）通过归一化尾状核体积评估的疾病严重程度之间的关联。 HD 患者的壳核中扩张的 PVS 体积百分比最大（左侧壳核：比值比 2.06 [95% 置信区间 (CI) 1.62–2.62]，HD 3.27% [95% CI 2.83–3.78] 对比对照组 1.62% [95% CI 1.32–1.97]，pfdr < 0.001；右侧壳核：与对照组相比，比值比为 1.66 [95% CI 1.33–2.08]，HD 3.43% [95% CI 2.94–4.01] 2.09% [95% CI 1.79–2.45]，pfdr < 0.001）和几个皮质下白质区域。扩张的PVS随着疾病的严重程度而增加。扩张的PVS的客观量化表明PVS负担很高，与疾病严重程度相关，并且可能影响鞘内施用（例如反义寡核苷酸）或实质内施用（例如细胞和基因疗法）的治疗的分布和成功。这项研究提供了 II 类证据，表明扩张的 PVS 增加与 HD 严重程度恶化相关。由于横截面设计，该研究被评为 II 级。
To quantify the percent volume of dilated perivascular space (PVS) in the subcortical forebrain in patients with early Huntington disease (HD) and to explore the relationship between PVS and disease severity. MRI scans were performed on 25 patients with HD and 23 healthy age-matched controls at Massachusetts General Hospital. The imaging data were analyzed with a novel algorithm to determine regional PVS volume. A fractional logistic regression analysis was used to quantify the association between regional percent PVS volume and (1) disease designation (HD or control) and (2) disease severity as assessed by normalized caudate volume. Patients with HD had the greatest percent volume of dilated PVS in the putamen (left putamen: odds ratio 2.06 [95% confidence interval (CI) 1.62–2.62], HD 3.27% [95% CI 2.83–3.78] vs controls 1.62% [95% CI 1.32–1.97], pfdr < 0.001; right putamen: odds ratio 1.66 [95% CI 1.33–2.08], HD 3.43% [95% CI 2.94–4.01] vs controls 2.09% [95% CI 1.79–2.45], pfdr < 0.001) and several subcortical white matter regions compared to controls. Dilated PVS increased with disease severity. The objective quantification of dilated PVS suggests that PVS burden is high, is associated with disease severity, and may affect the distribution and success of treatments administered either intrathecally such as antisense oligonucleotides or by intraparenchymal administration such as cell and gene therapies. This study provides Class II evidence that increased dilated PVS is associated with worse HD severity. The study is rated Class II because of the cross-sectional design.