Horiuchi Hiroshi, Saito Naoto, Kobayashi Seneki, Ota Hiroyoshi, Taketomi Tamotsu, Takaoka Kunio
Shinshu University, Asahi, Matsumoto, Japan.
Arthritis Rheum. 2002 Jul;46(7):1922-5. doi: 10.1002/art.10391.
Fabry's disease is a lipid storage disease caused by an X-linked hereditary deficiency of alpha-galactosidase. The enzymatic defect causes progressive deposition of ceramide trihexoside (CTH) in various tissues, leading to renal failure, premature myocardial infarction, and stroke, with a high rate of mortality in younger patients. Among the complications associated with Fabry's disease, a few cases involving avascular necrosis (AVN) of the femoral head have been reported. However, direct evidence of deposition of CTH in bone marrow in the femoral head has not been demonstrated. This report describes a 58-year-old man who underwent total hip arthroplasty for femoral head AVN associated with Fabry's disease. The accumulation of CTH was examined by chemical analysis of the sphingolipid extracted from the femoral head, using delayed-extraction matrix-assisted laser desorption ionization-time-of-flight mass spectrometry. This is the first report confirming the presence of CTH in the sphingolipid fraction from normal and necrotic bone of a patient with Fabry's disease.
法布里病是一种脂质贮积病,由X连锁遗传性α-半乳糖苷酶缺乏引起。酶缺陷导致神经酰胺三己糖苷(CTH)在各种组织中进行性沉积,从而导致肾衰竭、过早发生心肌梗死和中风,年轻患者死亡率很高。在与法布里病相关的并发症中,已有少数涉及股骨头缺血性坏死(AVN)的病例报道。然而,尚未证实CTH在股骨头骨髓中沉积的直接证据。本报告描述了一名58岁男性,因法布里病相关的股骨头AVN接受了全髋关节置换术。使用延迟提取基质辅助激光解吸电离飞行时间质谱法,通过对从股骨头提取的鞘脂进行化学分析,检测CTH的蓄积情况。这是首次证实法布里病患者正常和坏死骨的鞘脂部分中存在CTH的报告。