Martelli M, Saponaro A
Minerva Med. 1983 Oct 27;74(41):2429-38.
Marfan's disease is a hereditary condition (usually dominant) characterised by variously significant skeletal muscle, ocular, cardiac and above all respiratory alterations attributable to congenital disorder of the fibrous support proteins (particularly of collagen and elastin). Sporadic forms whose interpretation is uncertain, however may, also be observed. The exact nature of the biochemical error responsible for the syndrome, however, is not known. In the absence of fully indicative laboratory tests, diagnosis is based on recognition of he typical lesions and their systemic nature. Careful symptomatological examination of suspected subjects may lead to the detection of less common sites such as the respiratory system. Personal experience shows that it can also reveal clinically obsolete lesions, such as heart impairment discovered in some cases solely through elevation of the polycardiographic telediastolic index (in inverse relation to the pattern of the echocardiographic telediastolic volume), which is an expression of reduced ventricular compliance, and the presence of areas with a low thallium uptake, offering scintigraphic evidence of fibrosis replacing destroyed muscle fibres. Prognosis depends on the clinical expressiveness of the disease, i.e. the apparatuses involved and the extent of their damage. Heart alterations and their extent are undoubtedly an aggravating factor quoad vitam. The current position with regard to both drug management and possible surgical treatment is also discussed.
马凡氏综合征是一种遗传性疾病(通常为显性遗传),其特征是由于纤维支撑蛋白(特别是胶原蛋白和弹性蛋白)的先天性紊乱,导致骨骼肌、眼部、心脏尤其是呼吸系统出现不同程度的显著改变。然而,也可能观察到一些解释尚不明确的散发病例。然而,导致该综合征的生化错误的确切性质尚不清楚。由于缺乏完全具有指示性的实验室检查,诊断基于对典型病变及其全身性特征的识别。对疑似患者进行仔细的症状学检查可能会发现一些不太常见的部位,如呼吸系统。个人经验表明,检查还可能揭示临床上已被忽视的病变,例如在某些病例中仅通过多心电图舒张末期指数升高(与超声心动图舒张末期容积模式呈反比关系,这是心室顺应性降低的一种表现)发现的心脏损害,以及存在铊摄取低的区域,这为纤维化替代受损肌纤维提供了闪烁扫描证据。预后取决于疾病的临床表现,即受累的器官及其损害程度。心脏改变及其程度无疑是影响生命的一个加重因素。文中还讨论了药物治疗和可能的手术治疗的现状。