Department of Neurology, Friedrich-Alexander-Universität Erlangen-Nürnberg (FAU), Erlangen, Germany.
Department of Molecular Neurology, FAU, Schwabachanlage 6, 91054, Erlangen, Germany.
Orphanet J Rare Dis. 2022 Jul 29;17(1):301. doi: 10.1186/s13023-022-02451-1.
SPG11-linked hereditary spastic paraplegia is characterized by multisystem neurodegeneration leading to a complex clinical and yet incurable phenotype of progressive spasticity and weakness. Severe cognitive symptoms are present in the majority of SPG11 patients, but a systematic and multidimensional analysis of the neuropsychological phenotype in a larger cohort is lacking. While thinning of the corpus callosum is a well-known structural hallmark observed in SPG11 patients, the neuroanatomical pattern of cortical degeneration is less understood. We here aimed to integrate neuropsychological and brain morphometric measures in SPG11.
We examined the neuropsychological profile in 16 SPG11 patients using a defined neuropsychological testing battery. Long-term follow up testing was performed in 7 patients. Cortical and subcortical degeneration was analyzed using an approved, artificial intelligence based magnetic resonance imaging brain morphometry, comparing patients to established reference values and to matched controls.
In SPG11 patients, verbal fluency and memory as well as frontal-executive functions were severely impaired. Later disease stages were associated with a global pattern of impairments. Interestingly, reaction times correlated significantly with disease progression. Brain morphometry showed a significant reduction of cortical and subcortical parenchymal volume following a rostro-caudal gradient in SPG11. Whereas performance in memory tasks correlated with white matter damage, verbal fluency measures showed strong associations with frontal and parietal cortical volumes.
The present data will help define neuropsychological and imaging read out parameters in early as well as in advanced clinical stages for future interventional trials in SPG11.
SPG11 相关性遗传性痉挛性截瘫的特征是多系统神经退行性变,导致进行性痉挛和无力的复杂临床且不可治愈的表型。大多数 SPG11 患者存在严重的认知症状,但缺乏对更大队列中神经心理学表型的系统和多维分析。虽然胼胝体变薄是 SPG11 患者中众所周知的结构标志,但皮质退化的神经解剖模式了解较少。我们旨在整合 SPG11 中的神经心理学和大脑形态计量学测量。
我们使用定义明确的神经心理学测试包检查了 16 名 SPG11 患者的神经心理学概况。对 7 名患者进行了长期随访测试。使用经过批准的基于人工智能的磁共振成像脑形态计量学分析皮质和皮质下变性,将患者与既定的参考值和匹配的对照组进行比较。
在 SPG11 患者中,言语流畅性和记忆力以及额叶执行功能严重受损。晚期疾病阶段与整体损伤模式相关。有趣的是,反应时间与疾病进展显著相关。脑形态计量学显示 SPG11 患者的皮质和皮质下实质体积呈颅尾梯度显著减少。虽然记忆任务的表现与白质损伤相关,但言语流畅性测量与额叶和顶叶皮质体积有很强的关联。
目前的数据将有助于定义 SPG11 早期和晚期临床阶段的神经心理学和影像学读出参数,以用于未来的干预性试验。