Pomar Blanco P, San Román Carbajo J, Martín Villares C, Rodríguez Martín F, Paniagua J, Fernández Pello M, Tapia Risueño M
Servicio de ORL, Sacyl. Hospital Comarcal El Bierzo, León.
Acta Otorrinolaringol Esp. 2006 Feb;57(2):115-7. doi: 10.1016/s0001-6519(06)78672-1.
Anderson-Fabry disease in an inherited X-linked metabolic disorder involving glycosphingolipid metabolism. Few data are available regarding cochlear involvement. Clinical manifestations of Fabry disease appeared on the first decade of life. The prognosis of males with Fabry disease is serious and life expectancy is limited; clinical evolution of heterozygous females is clearly better. We report a family with Fabry disease in several members. The mother, already dead, had two child which have been examined in our department; the male, without a risk of ototoxicity, or acoustic trauma, has progressive hearing loss, tinnitus and dizinness sometimes; the daughter, without a history of deafness, shows unilateral hearing loss on high-tone frequencies. It is important to emphasize these data to those physicians expert in children with Fabry disease because early enzyme replacement therapy intervention should offer increased possibilities of regression of the disease.
安德森-法布里病是一种涉及糖鞘脂代谢的遗传性X连锁代谢紊乱疾病。关于耳蜗受累的数据很少。法布里病的临床表现出现在生命的第一个十年。男性法布里病患者的预后严重,预期寿命有限;杂合子女性的临床病程明显较好。我们报告了一个有多名成员患法布里病的家族。已去世的母亲有两个孩子在我们科室接受了检查;该男性没有耳毒性或声学创伤风险,有时会出现进行性听力损失、耳鸣和头晕;女儿没有耳聋病史,表现为高频单侧听力损失。向那些治疗法布里病患儿的专家强调这些数据很重要,因为早期酶替代疗法干预应该会增加疾病消退的可能性。