Tomizawa Yuji, Okuzumi Ayami, Shiotsuki Hiromi, Noda Kazuyuki, Hattori Nobutaka, Okuma Yasuyuki
Department of Neurology, Juntendo University Shizuoka Hospital, Japan.
Intern Med. 2015;54(19):2503-6. doi: 10.2169/internalmedicine.54.4437. Epub 2015 Oct 1.
A 76-year-old man was admitted to our hospital due to gait difficulty. Brain imaging indicated bilateral pulvinar lesions and moderate white matter lesions. The serum α-galactosidase A levels were measured for the differential diagnosis of bilateral pulvinar lesions and were found to be abnormally low. Therefore, the patient was suspected to have variant Fabry disease. A GLA mutation analysis showed the p.E66Q mutation, which is speculated to be a functional polymorphism rather than a disease-causing mutation of Fabry disease. Enzyme replacement therapy did not result in a marked improvement, however, the disease progression stopped.
一名76岁男性因步态困难入院。脑部影像学检查显示双侧丘脑枕部病变及中度白质病变。检测血清α-半乳糖苷酶A水平以鉴别双侧丘脑枕部病变,结果发现其异常低。因此,怀疑该患者患有变异型法布里病。GLA突变分析显示存在p.E66Q突变,推测这是一种功能性多态性而非法布里病的致病突变。酶替代疗法虽未带来显著改善,但疾病进展停止。