波多黎各一名 3 岁患者的高丙种球蛋白血症 D 和周期性发热综合征 (HIDS)。

Puerto Rico health sciences journal Pub Date : 2024-09-01
Abdiel J Alicea-Negrón, Marilyn Toledo-García, Marta Torres-Quiñones, Annette López-Martínez, Jose R González-Chávez
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引用次数: 0

摘要

甲羟戊酸激酶缺乏症是一种由甲羟戊酸激酶基因(MVK)突变引起的罕见常染色体隐性遗传病。根据基因突变的不同,这种缺乏症患者可表现出一系列罕见自身炎症性疾病中的任何一种,如高丙种球蛋白血症 D(高 IgD)伴周期性发热综合征和甲羟戊酸尿症。迄今为止,全世界已报道了约 300 例 MVK 基因突变病例。在本文中,我们介绍了一名来自波多黎各的 3 岁女性患者,她自出生一个月起就出现发热、关节痛和皮肤损伤,经基因检测证实,她的 MVK 基因存在复合杂合突变。根据她的病史和基因检测结果,她被诊断为高IgD伴周期性发热综合征。在接种水痘和麻疹-腮腺炎-风疹(MMR)疫苗后,她将接受白细胞介素-1ß拮抗剂卡那单抗(canakinumab)治疗。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Hypergammaglobulinemia D and Periodic Fever Syndrome (HIDS) in a 3-year-old Patient from Puerto Rico.

Mevalonate kinase deficiency is a rare autosomal recessive disease caused by mutations in the mevalonate kinase gene (MVK). Depending on the mutations, a patient with this deficiency can exhibit any one of a spectrum of rare autoinflammatory diseases, such as hypergammaglobulinemia D (hyper-IgD) with periodic fever syndrome and mevalonic aciduria. To date, approximately 300 cases with mutations in the MVK gene have been reported worldwide. Herein, we present a 3-year-old female from Puerto Rico with a history of fever, arthralgia, and skin lesions since her first month of age and who, upon genetic workup, was confirmed to have compound heterozygous mutations in the MVK gene. Given her medical history and the results of her genetic testing, she was diagnosed with hyper-IgD with periodic fever syndrome. She will be treated with canakinumab, an interleukin-1ß antagonist, after receiving the varicella and measles-mumps-rubella (MMR) vaccines.

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