Department of Hematology and Bone Marrow Transplantation, Rambam Health Care Campus, Haifa, Israel.
Semin Thromb Hemost. 2013 Nov;39(8):928-34. doi: 10.1055/s-0033-1357485. Epub 2013 Oct 15.
Gaucher disease (GD) is a lysosomal disorder caused by inherited deficiency of glucocerebrosidase (GCase), resulting in the accumulation of glucocerebroside in macrophages, termed "Gaucher cells," leading to multiorgan involvement, with hepatosplenomegaly, cytopenias, pulmonary hypertension, and skeletal complications. Various mutations, encoding the GCase gene, cause acute or chronic neuronopathic forms of the disease. The hallmark of GD is the macrophages infiltrating organs, bone marrow, and nervous system compromising their function by inflammation, infarcts, fibrosis, and neuronal damage. Coagulation abnormalities are frequent among GD patients due to reduced production and chronic consumption of coagulation factors. Splenic and bone infarcts often occur in GD patients, but hypercoagulability is not frequent. Detection of thrombophilic risk factors in GD patients may predict a more severe course of the disease. Clinical and genetic studies revealed an association between reduced GCase activity in carriers of GD mutations and GD patients and occurrence of Parkinson disease (PD) and showed that GCase gene mutations are risk factors for PD development. The mechanisms underlying the association of PD and GD are not yet elucidated and should be further explored, particularly the potential involvement of inflammation and coagulation in the neurovascular unit.
戈谢病(GD)是一种溶酶体贮积病,由葡糖脑苷脂酶(GCase)遗传性缺乏引起,导致葡糖脑苷脂在巨噬细胞(称为“戈谢细胞”)中积累,从而导致多器官受累,出现肝脾肿大、细胞减少症、肺动脉高压和骨骼并发症。各种突变导致疾病的急性或慢性神经病变形式。GD 的标志是巨噬细胞浸润器官、骨髓和神经系统,通过炎症、梗死、纤维化和神经元损伤来损害其功能。由于凝血因子生成减少和慢性消耗,戈谢病患者常出现凝血异常。脾梗死和骨梗死在 GD 患者中经常发生,但高凝状态并不常见。在 GD 患者中检测到血栓形成倾向的危险因素可能预示着疾病更严重的病程。临床和遗传研究表明,GD 突变携带者和 GD 患者的 GCase 活性降低与帕金森病(PD)的发生有关,并且表明 GCase 基因突变是 PD 发展的危险因素。PD 和 GD 之间关联的机制尚未阐明,应进一步探讨,特别是炎症和凝血在神经血管单元中的潜在作用。