Debette Stéphanie, Germain Dominique P
Department of Epidemiology and Public Health, Raymond Poincaré Hospital, Garches, France; INSERM Unit U708, Pitié-Salpêtrière Hospital, Paris, France; University of Versailles - St Quentin en Yvelines, Versailles, France.
University of Versailles - St Quentin en Yvelines, Versailles, France; Division of Medical Genetics, National Referral Center for Fabry Disease and Inherited Disorders of Connective Tissue, CHU Raymond Poincaré, Garches, France.
Handb Clin Neurol. 2014;119:565-76. doi: 10.1016/B978-0-7020-4086-3.00037-0.
Inherited disorders of connective tissue are single gene disorders affecting structure or function of the connective tissue. Neurological manifestations are classic and potentially severe complications of many such disorders. The most common neurological manifestations are cerebrovascular. Ischemic stroke is a classic complication of vascular Ehlers-Danlos syndrome (type IV), homocystinuria, and arterial tortuosity syndrome, and may occasionally be seen in Marfan syndrome and pseudoxanthoma elasticum with distinct underlying mechanisms for each disease. Vascular Ehlers-Danlos syndrome can also lead to cervical artery dissection (with or without ischemic stroke), carotid-cavernous fistula, intracranial dissections and aneurysms potentially causing subarachnoid or intracerebral hemorrhage, and arterial rupture. Other neurological manifestations include nerve root compression and intracranial hypotension due to dural ectasia in Marfan and Loeys-Dietz syndrome, spinal cord compression in osteogenesis imperfecta, and mucopolysaccharidosis type I and VI, carpal tunnel syndrome in mucopolysaccharidosis type I, II, and VI. Impaired mental development can be observed in homocystinuria, mucopolysaccharidosis type II, and the severe form of mucopolysaccharidosis type I. For the neurologist, being aware of these complications and of the diagnostic criteria for inherited connective tissue disorders is important since neurological complications can be the first manifestation of the disease and because caution may be warranted for the management of these patients.
遗传性结缔组织疾病是影响结缔组织结构或功能的单基因疾病。神经学表现是许多此类疾病的典型且可能严重的并发症。最常见的神经学表现是脑血管方面的。缺血性中风是血管型埃勒斯-当洛综合征(IV型)、同型胱氨酸尿症和动脉迂曲综合征的典型并发症,在马方综合征和弹性假黄瘤中偶尔也可见到,每种疾病有不同的潜在发病机制。血管型埃勒斯-当洛综合征还可导致颈动脉夹层(伴或不伴缺血性中风)、颈动脉海绵窦瘘、颅内夹层和动脉瘤,可能引起蛛网膜下腔或脑内出血以及动脉破裂。其他神经学表现包括马方综合征和洛伊斯-迪茨综合征中因硬脑膜扩张导致的神经根受压和颅内低压、成骨不全、黏多糖贮积症I型和VI型中的脊髓受压、黏多糖贮积症I型、II型和VI型中的腕管综合征。在同型胱氨酸尿症、黏多糖贮积症II型以及黏多糖贮积症I型的严重形式中可观察到智力发育受损。对于神经科医生而言,了解这些并发症以及遗传性结缔组织疾病的诊断标准很重要,因为神经并发症可能是疾病的首发表现,而且在治疗这些患者时可能需要谨慎行事。