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血影蛋白基因(HJV)新突变致青少年血色病伴发胰岛素依赖型糖尿病、继发性甲状腺功能减退和性腺功能减退

Novel Mutation in the Hemojuvelin Gene (HJV) in a Patient with Juvenile Hemochromatosis Presenting with Insulin-dependent Diabetes Mellitus, Secondary Hypothyroidism and Hypogonadism.

机构信息

Endocrinology Unit, University Hospital of the Federal University of Maranhão (HUUFMA), São Luís, Brazil.

Research Group in Clinical and Molecular Endocrinology and Metabology (ENDOCLIM), São Luís, Brazil.

出版信息

Am J Case Rep. 2020 Apr 24;21:e923108. doi: 10.12659/AJCR.923108.

DOI:10.12659/AJCR.923108
PMID:32327622
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7200091/
Abstract

BACKGROUND Juvenile hemochromatosis is a rare genetic disease that leads to intense iron accumulation. The disease onset usually occurs before the third decade of life and causes severe dysfunction in various organs. The most classical clinical findings are hypogonadotropic hypogonadism, cardiomyopathy, liver fibrosis, glycemic changes, arthropathy and skin pigmentation. However, secondary hypothyroidism is not reported in these patients. Juvenile hemochromatosis has an autosomal recessive inheritance and might be type 2A or type 2B, due to mutation in either the hemojuvelin gene (HJV) or hepcidin antimicrobial peptide (HAMP) gene. CASE REPORT A 26-year-old female patient was admitted with a recent history of diabetic ketoacidosis. Three months after that admission, she presented with arthralgia, diffuse abdominal pain, adynamia, hair loss, darkening of the skin and amenorrhea. Severe iron overload was found and findings in the hepatic biopsy were compatible with hemochromatosis. An upper abdominal magnetic resonance imaging (MRI) showed iron deposition in the liver and pancreas and pituitary MRI exhibited accumulation on the anterior pituitary. After 16 months the patient presented with dyspnea and lower limb edema, and cardiac MRI indicated iron deposition in the myocardium. The patient was diagnosed with juvenile hemochromatosis presenting with hypogonadotropic hypogonadism, cardiomyopathy, insulin-dependent diabetes mellitus, and secondary hypothyroidism. A novel homozygous mutation, c.697delC, in the HJV gene was detected. CONCLUSIONS We describe for the first time a severe and atypical case of juvenile hemochromatosis type 2A presenting classical clinical features, as well as secondary hypothyroidism resulting from a novel mutation in the HJV gene.

摘要

背景

青少年血色病是一种罕见的遗传性疾病,导致铁的大量积累。发病通常发生在 30 岁之前,导致多个器官严重功能障碍。最典型的临床表现是促性腺激素低下性性腺功能减退症、心肌病、肝纤维化、血糖变化、关节炎和皮肤色素沉着。然而,这些患者并没有报道继发性甲状腺功能减退症。青少年血色病为常染色体隐性遗传,可能是 2A 型或 2B 型,由于珠蛋白生成素基因(HJV)或抗菌肽基因(HAMP)突变。

病例报告

一名 26 岁女性患者因近期糖尿病酮症酸中毒入院。入院 3 个月后,出现关节痛、弥漫性腹痛、乏力、脱发、皮肤变黑和闭经。发现严重的铁过载,肝活检结果与血色病相符。上腹部磁共振成像(MRI)显示肝脏和胰腺铁沉积,垂体 MRI 显示前垂体有堆积。16 个月后,患者出现呼吸困难和下肢水肿,心脏 MRI 显示心肌铁沉积。患者被诊断为青少年血色病,表现为促性腺激素低下性性腺功能减退症、心肌病、胰岛素依赖型糖尿病和继发性甲状腺功能减退症。检测到 HJV 基因中的新型纯合突变 c.697delC。

结论

我们首次描述了一例严重和非典型的 2A 型青少年血色病病例,表现为典型的临床表现,以及由于 HJV 基因的新型突变导致的继发性甲状腺功能减退症。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/77d2/7200091/962b9e7f3a3b/amjcaserep-21-e923108-g005.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/77d2/7200091/25d4d192e5ba/amjcaserep-21-e923108-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/77d2/7200091/241ff1a32515/amjcaserep-21-e923108-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/77d2/7200091/4c271ab6fe2c/amjcaserep-21-e923108-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/77d2/7200091/364ad30971f9/amjcaserep-21-e923108-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/77d2/7200091/962b9e7f3a3b/amjcaserep-21-e923108-g005.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/77d2/7200091/25d4d192e5ba/amjcaserep-21-e923108-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/77d2/7200091/241ff1a32515/amjcaserep-21-e923108-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/77d2/7200091/4c271ab6fe2c/amjcaserep-21-e923108-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/77d2/7200091/364ad30971f9/amjcaserep-21-e923108-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/77d2/7200091/962b9e7f3a3b/amjcaserep-21-e923108-g005.jpg

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