Brandt Alexander U, Meinert-Bohn Elena, Rinnenthal Jan Leo, Zimmermann Hanna, Mikolajczak Janine, Oberwahrenbrock Timm, Papazoglou Sebastian, Pfüller Caspar F, Schinzel Johann, Tackenberg Björn, Paul Friedemann, Hahn Katrin, Bellmann-Strobl Judith
NeuroCure Clinical Research Center, Charité -Universitätsmedizin Berlin, Berlin, Germany.
Institute of Neuropathology, Charité -Universitätsmedizin Berlin, Berlin, Germany.
PLoS One. 2016 Oct 17;11(10):e0164617. doi: 10.1371/journal.pone.0164617. eCollection 2016.
The PMP22 gene encodes a protein integral to peripheral myelin. Its deletion leads to hereditary neuropathy with liability to pressure palsies (HNPP). PMP22 is not expressed in the adult central nervous system, but previous studies suggest a role in CNS myelin development. The objective of this study was to identify potential structural and functional alterations in the afferent visual system in HNPP patients.
Twenty HNPP patients and 18 matched healthy controls (HC) were recruited in a cross-sectional study. Participants underwent neurological examination including visual acuity, visual evoked potential (VEP) examination, optical coherence tomography (OCT), and magnetic resonance imaging with calculation of brain atrophy, regarding grey and white matter, and voxel based morphometry (VBM), in addition answered the National Eye Institute's 39-item Visual Functioning Questionnaire (NEI-VFQ). Thirteen patients and 6 HC were additionally examined with magnetic resonance spectroscopy (MRS).
All patients had normal visual acuity, but reported reduced peripheral vision in comparison to HC in the NEI-VFQ (p = 0.036). VEP latency was prolonged in patients (P100 = 103.7±5.7 ms) in comparison to healthy subjects (P100 = 99.7±4.2 ms, p = 0.007). In OCT, peripapillary retinal nerve fiber layer thickness RNFL was decreased in the nasal sector (90.0±15.5 vs. 101.8±16.5, p = 0.013), and lower nasal sector RNFL correlated with prolonged VEP latency (Rho = -0.405, p = 0.012). MRS revealed reduced tNAA (731.4±45.4 vs. 814.9±62.1, p = 0.017) and tCr (373.8±22.2 vs. 418.7±31.1, p = 0.002) in the visual cortex in patients vs. HC. Whole brain volume, grey and white matter volume, VBM and metabolites in a MRS sensory cortex control voxel did not differ significantly between patients and HC.
PMP22 deletion leads to functional, metabolic and macro-structural alterations in the afferent visual system of HNPP patients. Our data suggest a functional relevance of these changes for peripheral vision, which warrants further investigation and confirmation.
PMP22基因编码一种外周髓鞘不可或缺的蛋白质。其缺失会导致遗传性压力易感性神经病(HNPP)。PMP22在成体中枢神经系统中不表达,但先前的研究表明其在中枢神经系统髓鞘发育中发挥作用。本研究的目的是确定HNPP患者传入视觉系统中潜在的结构和功能改变。
在一项横断面研究中招募了20名HNPP患者和18名匹配的健康对照(HC)。参与者接受了神经系统检查,包括视力、视觉诱发电位(VEP)检查、光学相干断层扫描(OCT)以及用于计算脑萎缩(涉及灰质和白质)的磁共振成像和基于体素的形态测量(VBM),此外还回答了美国国立眼科研究所的39项视觉功能问卷(NEI-VFQ)。另外13名患者和6名HC接受了磁共振波谱(MRS)检查。
所有患者视力正常,但与健康对照相比,在NEI-VFQ中报告周边视力下降(p = 0.036)。与健康受试者相比,患者的VEP潜伏期延长(患者P100 = 103.7±5.7毫秒,健康受试者P100 = 99.7±4.2毫秒,p = 0.007)。在OCT检查中,患者视乳头周围视网膜神经纤维层(RNFL)厚度在鼻侧扇形区域降低(90.0±15.5 vs. 101.8±16.5,p = 0.013),并且鼻侧扇形区域较低的RNFL与延长的VEP潜伏期相关(Rho = -0.405,p = 0.012)。MRS显示,与健康对照相比,患者视觉皮层中的总N-乙酰天门冬氨酸(tNAA)降低(731.4±45.4 vs. 814.9±62.1,p = 0.017),总肌酸(tCr)降低(373.8±22.2 vs. 418.7±31.1,p = 0.002)。患者和健康对照之间全脑体积、灰质和白质体积、VBM以及MRS感觉皮层对照体素中的代谢物没有显著差异。
PMP22缺失导致HNPP患者传入视觉系统出现功能、代谢和宏观结构改变。我们的数据表明这些变化与周边视力存在功能相关性。这值得进一步研究和证实。