Chan B, Adam D N
McMaster University, Hamilton, ON, Canada.
Baywood Dermatology, Ajax, ON, Canada; CCA Medical Research, Ajax, ON, Canada; St. Michael's Hospital, Toronto, ON, Canada; Division of Dermatology, Department of Medicine, University of Toronto, Toronto, ON, Canada; Probity Medical Research, Waterloo, ON, Canada.
Skin Therapy Lett. 2018 Mar;23(2):4-6.
Fabry disease (FD) is an X-linked lysosomal storage disease. A lack of alpha-galactosidase activity results in the accumulation of globotriaosylceramide in cells of various systems, leading to multi-systemic effects. The cutaneous hallmark of FD is a specific distribution of angiokeratoma. Other common symptoms include cornea verticillata, acroparesthesia, and sweating abnormalities. FD-specific symptoms, history, as well as examination of angiokeratoma can assist in the differential diagnosis. Enzyme replacement therapy is the current mainstay of treatment.
法布里病(FD)是一种X连锁溶酶体贮积病。α-半乳糖苷酶活性缺乏导致球三糖神经酰胺在多个系统的细胞中蓄积,从而产生多系统效应。FD的皮肤特征是血管角质瘤的特定分布。其他常见症状包括角膜涡状浑浊、肢端感觉异常和出汗异常。FD的特异性症状、病史以及血管角质瘤检查有助于鉴别诊断。酶替代疗法是目前的主要治疗方法。