Department of Pediatrics, Division of Genetics and Metabolism, University of Texas Southwestern Medical Center, Dallas, Texas.
Am J Med Genet A. 2019 Aug;179(8):1415-1419. doi: 10.1002/ajmg.a.61205. Epub 2019 May 22.
Incontinentia Pigmenti (IP; MIM 308300) is an X-linked dominant genodermatosis caused by pathogenic variant in IKBKG. The phenotype in adults is poorly described compared to that in children. Questionnaire survey of 99 affected women showed an age at diagnosis from newborn to 41 years, with 53 diagnosed by 6 months of age and 30 as adults. Stage I, II, and III lesions persisted in 16%, 17%, and 71%, respectively, of those who had ever had them. IP is allelic to two forms of ectodermal dysplasia. Many survey respondents reported hypohidrosis and/or heat intolerance and most had Stage IV findings. This suggests that "Stage IV" may be congenitally dysplastic skin that becomes more noticeable with maturity. Fifty-one had dentures or implants with 26 having more invasive jaw or dental surgery. Half had wiry or uncombable hair. Seventy-three reported abnormal nails with 27 having long-term problems. Cataracts and retinal detachment were the reported causes of vision loss. Four had microphthalmia. Respondents without genetic confirmation of IP volunteered information suggesting more involved phenotype or possibly misassigned diagnosis. Ascertainment bias likely accounts for the low prevalence of neurocognitive problems in the respondents.
遗传性皮肤病(IP;MIM 308300)是一种 X 连锁显性遗传的皮肤病,由 IKBKG 的致病变异引起。与儿童相比,成人的表型描述较差。对 99 名受影响女性的问卷调查显示,发病年龄从新生儿到 41 岁不等,其中 53 名在 6 个月大时被诊断出,30 名在成年后被诊断出。曾患有该病的患者中,分别有 16%、17%和 71%的患者仍存在 I 期、II 期和 III 期病变。IP 与两种形式的外胚层发育不良相关。许多调查受访者报告有少汗症和/或不耐热,大多数人有 IV 期表现。这表明“IV 期”可能是先天性发育不良的皮肤,随着成熟变得更加明显。51 人有义齿或植入物,其中 26 人进行了更具侵入性的颌骨或牙科手术。一半人有卷曲或难以梳理的头发。73 人报告指甲异常,其中 27 人有长期问题。白内障和视网膜脱离是视力丧失的报告原因。有 4 人患有小眼球。没有 IP 遗传确认的受访者自愿提供信息,表明存在更严重的表型或可能存在误诊。确定偏差可能是受访者中神经认知问题患病率较低的原因。