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男性(XY)婴儿的色素失禁症,长期随访超过 8 年。

Incontinentia pigmenti in a male (XY) infant with long-term follow up over 8 years.

机构信息

Department of Dermatology, Hirosaki University Graduate School of Medicine, Hirosaki, Japan.

Nomura Dermatology Clinic, Hirosaki, Japan.

出版信息

J Dermatol. 2018 Jan;45(1):100-103. doi: 10.1111/1346-8138.14002. Epub 2017 Aug 9.

Abstract

Incontinentia pigmenti (IP) is an X-linked genodermatosis affecting the skin and other sites, including the teeth, nails, hair, eyes and nervous system defects in female patients. Generally lethal in males, there are only a few known cases of males surviving this condition. Nuclear factor (NF)-κB essential modulator (NEMO), also known as inhibitor of kappa light polypeptide gene enhancer in B cells, kinase gamma (IKBKG), constitutes an essential activator of NF-κB. Over 80% of female patients with IP carry a common deletion mutation involving exons 4-10 of the IKBKG/NEMO gene. We present the case of a male infant (XY) with IP with no concomitant complications. Polymerase chain reaction (PCR) assay showed that the exon 4-10 deletion band was significantly stronger in the skin sample than in blood. Subsequently, long-range PCR was performed periodically to confirm the spontaneous regression of mutant cells from his blood. Over a period of 6 years, the 2.6-kb mutant band gradually became weaker, but we did not confirm complete regression. Our patient was a healthy, 8-year-old male child with no complications despite the presence of a 2.6-kb mutant band in his blood. Further follow up is necessary to assess for complications that may develop later.

摘要

遗传性皮肤病(IP)是一种 X 连锁的皮肤遗传病,还会影响牙齿、指甲、毛发、眼睛和神经系统等其他组织。女性患者通常致命,只有少数已知的男性存活病例。核因子(NF)-κB 必需调节剂(NEMO),又称 B 细胞κ轻链增强子核因子抑制物激酶γ(IKBKG),是 NF-κB 的必需激活剂。超过 80%的 IP 女性患者携带涉及 IKBKG/NEMO 基因外显子 4-10 的常见缺失突变。我们报告了一例 IP 伴发无其他并发症的男性婴儿(XY)。聚合酶链反应(PCR)检测显示,皮肤样本中外显子 4-10 缺失带明显强于血液。随后,定期进行长距离 PCR 以确认其血液中突变细胞的自发消退。在 6 年期间,2.6kb 的突变带逐渐减弱,但我们没有确认完全消退。尽管我们的患者血液中存在 2.6kb 的突变带,但他是一个 8 岁的健康男性,没有任何并发症。需要进一步随访以评估可能以后出现的并发症。

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