Plauchu H, de Chadarévian J P, Bideau A, Robert J M
Service of Clinical Genetics, Hôtel-Dieu Hospital, Lyon, France.
Am J Med Genet. 1989 Mar;32(3):291-7. doi: 10.1002/ajmg.1320320302.
We report the results of a comprehensive and systematic clinical study of 324 patients with hereditary hemorrhagic telangiectasia, selected from a total of 1,270 cases recruited by epidemiological survey. In 94% of the cases, familial occurrence suggested autosomal dominant inheritance; maximum penetrance for at least one manifestation was 97%. Epistaxis was reported by 96% of the patients and, in more than 50%, developed before age 20. Heavy and frequent bleeding occurred mainly in middle-aged patients. Telangiectasia was documented in 74% of cases, half of whom were younger than 30 years. The frequency of involvement of the hands and wrists was 41%, and for the face, 33%. Visceral involvement was present in 25% of patients, with affected lungs and CNS in the young and gastrointestinal tract and liver in older patients. Symptomatic urinary tract involvement was seen in only two/324 patients. Involvement of other internal sites was not observed.
我们报告了对324例遗传性出血性毛细血管扩张症患者进行的全面系统临床研究结果,这些患者是从通过流行病学调查招募的1270例患者中挑选出来的。在94%的病例中,家族性发病提示常染色体显性遗传;至少一种表现的最大外显率为97%。96%的患者有鼻出血症状,其中超过50%在20岁之前发病。严重且频繁的出血主要发生在中年患者中。74%的病例有毛细血管扩张记录,其中一半患者年龄小于30岁。手部和腕部受累的发生率为41%,面部为33%。25%的患者有内脏受累,年轻患者的受累部位为肺和中枢神经系统,老年患者为胃肠道和肝脏。仅在2/324例患者中观察到有症状的泌尿道受累。未观察到其他内部部位受累。