Green J S, Bowmer M I, Johnson G J
CMAJ. 1986 Jan 15;134(2):133-8, 146.
Von Hippel-Lindau disease, an autosomal dominant condition with complete penetrance, has been recognized in a large family that originated in Newfoundland but has some members who live in New Brunswick and Ontario. A collaborative investigation was begun in 1982 to document the number of affected members and the extent of their disease and to improve management of the disease. The condition has been documented in 38 members of the family, 28 living and 10 dead. The most common manifestations are retinal angioma (present in 60% of the gene carriers) and pheochromocytoma (present in 53%). Of the 28 living affected members 14 had been identified before the study began. Only 3 of the 14 patients in whom the disease was subsequently diagnosed presented with symptoms; in the remaining 11 the condition was detected by routine screening. Overall the mean age at the time of diagnosis was 23 years; in the 21 affected members of the fourth generation it was 18 years. The authors outline a regimen of regular screening for members at risk that has evolved as a result of their experience with this family.
冯·希佩尔-林道病是一种具有完全外显率的常染色体显性遗传病,在一个起源于纽芬兰但有些成员居住在新不伦瑞克和安大略的大家族中被确认。1982年开始了一项合作研究,以记录受影响成员的数量及其疾病程度,并改善对该疾病的管理。该疾病已在这个家族的38名成员中得到记录,其中28名在世,10名已去世。最常见的表现是视网膜血管瘤(在60%的基因携带者中出现)和嗜铬细胞瘤(在53%的患者中出现)。在28名在世的受影响成员中,有14名在研究开始前就已被确认。在随后被诊断出患有该疾病的14名患者中,只有3名出现了症状;其余11名是通过常规筛查发现病情的。总体而言,诊断时的平均年龄为23岁;在第四代的21名受影响成员中,平均年龄为18岁。作者概述了一种针对有患病风险成员的定期筛查方案,该方案是根据他们对这个家族的研究经验制定的。