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新生儿期起病的遗传性粪卟啉病:遗传性粪卟啉病的一种新变体

Neonatal-Onset Hereditary Coproporphyria: A New Variant of Hereditary Coproporphyria.

作者信息

Hasegawa Kosei, Tanaka Hiroyuki, Yamashita Miho, Higuchi Yousuke, Miyai Takayuki, Yoshimoto Junko, Okada Ayumi, Suzuki Norihiro, Iwatsuki Keiji, Tsukahara Hirokazu

机构信息

Department of Pediatrics, Okayama University Hospital, Shikatacho 2-5-1, Kita-ku, Okayama, 700-8558, Japan.

Department of Pediatrics, Okayama Saiseikai General Hospital, Okayama, Japan.

出版信息

JIMD Rep. 2017;37:99-106. doi: 10.1007/8904_2017_20. Epub 2017 Mar 28.

Abstract

Genetic mutation of the coproporphyrinogen oxidase (CPOX) gene causes either hereditary coproporphyria (HCP) or harderoporphyria. HCP, a rare hepatic porphyria, causes acute attacks after puberty and rarely accompanies cutaneous symptoms. In contrast, harderoporphyria is an erythropoietic porphyria that represents photosensitivity and hemolytic anemia from the neonatal period. In patients with harderoporphyria, the p.Lys404Glu mutation is found in the homozygous or compound heterozygous state with another mutation, and a marked increase in harderoporphyrin is observed. This report describes a neonate with symptoms of erythropoietic harderoporphyria (photosensitivity of the skin, hemolytic anemia, and jaundice). However, the pattern of porphyrin metabolites of feces was consistent with that of typical HCP, not of harderoporphyria. We found a heterozygous, novel, four-base pair deletion in exon 7 of the CPOX gene, although other mutations including the p.Lys404Glu mutation in CPOX were not found. By unknown etiology, our patient had accompanying adrenocortical insufficiency and 46, XY disorders of sex development. Based on genetic mutation of the CPOX gene and information from a previous similar case report, we consider that neonatal-onset HCP is a variant of HCP.

摘要

粪卟啉原氧化酶(CPOX)基因突变可导致遗传性粪卟啉病(HCP)或重型卟啉病。HCP是一种罕见的肝性卟啉病,青春期后会引发急性发作,很少伴有皮肤症状。相比之下,重型卟啉病是一种红细胞生成性卟啉病,从新生儿期就表现出光敏性和溶血性贫血。在重型卟啉病患者中,p.Lys404Glu突变以纯合子或复合杂合子状态与另一种突变同时存在,且观察到重型卟啉明显增加。本报告描述了一名患有红细胞生成性重型卟啉病症状(皮肤光敏性、溶血性贫血和黄疸)的新生儿。然而,粪便中卟啉代谢物的模式与典型的HCP一致,而非重型卟啉病。我们在CPOX基因外显子7中发现了一个杂合的、新的四碱基对缺失,尽管未发现包括CPOX基因中的p.Lys404Glu突变在内的其他突变。由于病因不明,我们的患者伴有肾上腺皮质功能不全和46, XY性发育障碍。基于CPOX基因的基因突变以及之前一份类似病例报告的信息,我们认为新生儿期发病的HCP是HCP的一种变体。

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