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迟发性庞贝病(LOPD)患者颅内动脉瘤的处理。

Intracranial aneurysm management in patients with late-onset Pompe disease (LOPD).

机构信息

Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy.

Neuroradiology Unit Department of Biomedical Sciences and Morphological and Functional Images, University of Messina, Via Consolare Valeria 1, 98100, Messina, Italy.

出版信息

Neurol Sci. 2021 Jun;42(6):2411-2419. doi: 10.1007/s10072-020-04819-2. Epub 2020 Oct 17.

DOI:10.1007/s10072-020-04819-2
PMID:33067680
Abstract

Pompe disease is a rare hereditary metabolic disorder caused by α-glucosidase (GAA) deficiency. The late-onset form of the disease (LOPD) is considered a multisystemic disorder which could involve vascular system with cerebrovascular abnormalities such as intracranial aneurysms or dolichoectasia. Intracranial aneurysm rupture may represent a life-threatening emergency. A possible treatment of unruptured intracranial aneurysms (UIAs) should consider both aneurysm-related (aneurysmal size, shape, localization, numbers and hemodynamic factors) and patient-related risk factors (patient's age and sex, hypertension, smoke exposure). Moreover, UIAs management of LOPD patients needs also to take into account the altered blood vessels integrity and elasticity, whose consistency is likely weakened by the deficient GAA activity as a further potential risk factor. We herein present our approach for of UIAs management in three patients with LOPD. Among them, only one patient with a left saccular UIA of the anterior communicating artery, after careful consideration of risk factors, underwent the endovascular treatment. The other two patients were scheduled for a 1-year follow-up, according to radiological, clinical, and risk evaluation features. Finally, we would like to suggest some general recommendations for UIAs management. In particular, if no risk factors are identified, a cautious yearly follow-up is suggested; otherwise, if risk factors are present, endovascular treatment should be considered.

摘要

庞贝病是一种罕见的遗传性代谢紊乱疾病,由α-葡萄糖苷酶(GAA)缺乏引起。疾病的迟发性形式(LOPD)被认为是一种多系统疾病,可能涉及血管系统,出现如颅内动脉瘤或梭形动脉瘤等脑血管异常。颅内动脉瘤破裂可能是危及生命的紧急情况。对于未破裂的颅内动脉瘤(UIAs)的可能治疗应同时考虑与动脉瘤相关的因素(动脉瘤大小、形状、位置、数量和血流动力学因素)和与患者相关的危险因素(患者年龄和性别、高血压、吸烟暴露)。此外,LOPD 患者的 UIAs 管理还需要考虑到血管完整性和弹性的改变,由于 GAA 活性缺乏,其一致性可能会减弱,这是另一个潜在的危险因素。我们在此介绍了我们对 3 例 LOPD 患者 UIAs 管理的方法。其中,只有 1 例患者存在前交通动脉左侧囊状 UIA,在仔细考虑了危险因素后,进行了血管内治疗。另外 2 例患者根据影像学、临床和风险评估特征,安排了为期 1 年的随访。最后,我们想提出一些 UIAs 管理的一般建议。特别是,如果没有发现风险因素,建议谨慎的每年随访;否则,如果存在风险因素,则应考虑血管内治疗。

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The impact of Pompe disease on smooth muscle: a review.庞贝病对平滑肌的影响:综述
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Central nervous system involvement in late-onset Pompe disease: clues from neuroimaging and neuropsychological analysis.迟发性庞贝病中枢神经系统受累:神经影像学和神经心理学分析的线索。
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Relationship between intracranial aneurysms and the severity of autosomal dominant polycystic kidney disease.颅内动脉瘤与常染色体显性多囊肾病严重程度之间的关系。
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Risk Factors for the Rupture of Bifurcation Intracranial Aneurysms Using CT Angiography.使用CT血管造影术分析颅内分叉动脉瘤破裂的危险因素
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