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中国家族性中枢性性早熟伴高尿酸血症与 DLK1 基因突变的复发性相关:病例报告及文献复习。

Chinese familial central precocious puberty with hyperuricemia due to recurrent DLK1 mutation: Case report and review of the literature.

机构信息

Department of Pediatric endocrinology, Quanzhou Women's and Children's Hospital, Quanzhou, China.

Department of Pediatric Surgery, Quanzhou Women's and Children's Hospital, Quanzhou, China.

出版信息

Mol Genet Genomic Med. 2022 Dec;10(12):e2087. doi: 10.1002/mgg3.2087. Epub 2022 Nov 9.

Abstract

BACKGROUND

Central precocious puberty (CPP) is a precocious puberty due to premature activation of the hypothalamic-pituitary-gonadal axis (HPG). MKRN3 defects are well-known causes of CPP, while DLK1 mutations were recently identified in a few patients with CPP.

METHODS

The study was approved by the Institutional Review and the scientific committee of the hospital. The clinical data were collected. Whole-exome sequencing (WES) was performed to detect causative variants. Key words 'DLK1', 'MKRN3', and "central precocious puberty" were used for literature search in PubMed, Google Scholar, HGMD, and OMIM databases.

RESULTS

The patient, a male, whose puberty began before age nine, had significant metabolic abnormalities including overweight, hyperlipidemia, and hyperuricemia. WES detected a recurrent frame-shift mutation, NM_003836.5:c.479delC(p.P160fs*50) in DLK1 in the patient and his father.

CONCLUSION

The familial DLK1-CPP was identified in China for the first time, which supported that short stature is predicted in patients with CPP without GnRHa treatment. Therefore, we recommend that children with DLK1-CPP should be treated as early as possible to improve adult height. The patient in this study had persistent hyperuricemia, further suggests that this antiadipogenic factor represents a link between reproduction and metabolism.

摘要

背景

中枢性性早熟(CPP)是由于下丘脑-垂体-性腺轴(HPG)过早激活引起的性早熟。MKRN3 缺陷是 CPP 的已知原因,而 DLK1 突变最近在少数 CPP 患者中被发现。

方法

本研究经机构审查委员会和医院科学委员会批准。收集临床资料。进行外显子组测序(WES)以检测致病变异。在 PubMed、Google Scholar、HGMD 和 OMIM 数据库中使用关键字“DLK1”、“MKRN3”和“中枢性性早熟”进行文献检索。

结果

该患者为男性,性早熟发生在 9 岁之前,存在明显的代谢异常,包括超重、血脂异常和高尿酸血症。WES 检测到患者及其父亲均存在 DLK1 中的重复移码突变 NM_003836.5:c.479delC(p.P160fs*50)。

结论

在中国首次鉴定出家族性 DLK1-CPP,这支持了未经 GnRHa 治疗的 CPP 患者存在预测身高矮小的情况。因此,我们建议尽早对患有 DLK1-CPP 的儿童进行治疗,以改善成年身高。本研究中的患者持续存在高尿酸血症,进一步表明这种抗脂肪生成因子是生殖与代谢之间的联系。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/cab5/9747546/4ab8bdbd17aa/MGG3-10-e2087-g001.jpg

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