SLC29A3变异、中性粒细胞皮肤病和高铁蛋白血症模拟沙特儿童系统性青少年特发性关节炎:1例报告。

IF 2.2 Q3 RHEUMATOLOGY
Shahad Alansari, Alhanouf Alsaleem, Tariq Alzaid, Maad Galal, Noura Alyahya, Sulaiman M Al-Mayouf
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引用次数: 1

摘要

SLC29A3的遗传缺陷导致广泛的综合征性组织细胞增多症,包括H综合征。SLC29A3变异的患者通常有色素沉着、多毛、肝脾肿大、感音神经性听力损失、糖尿病和性腺功能减退。在这里,我们确定了一个新的表现型,在一个女孩的临床和实验室发现类似的系统性少年关节炎和高铁蛋白血症。外显子组测序鉴定出SLC29A3的纯合子变异(NM_018344.5: c.707C>T [p.T236M])。我们的患者没有表现出slc29a3相关疾病广谱的主要特征。在开始使用白介素-1阻断(Anakinra)后,她的临床和实验室表现均有显著改善。最近的研究表明,slc29a3相关疾病伴随着自身炎症和自身免疫,这是由于炎症体途径过度活跃,这很可能是由线粒体和溶酶体功能障碍引起的。因此,我们的发现可能会扩展SLC29A3变异的表型特征。SLC29A3变异和全身性炎症患者可能受益于白细胞介素-1阻断作为治疗选择。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

The SLC29A3 variant, neutrophilic dermatosis, and hyperferritinemia imitate systemic juvenile idiopathic arthritis in a Saudi child: a case report.

The SLC29A3 variant, neutrophilic dermatosis, and hyperferritinemia imitate systemic juvenile idiopathic arthritis in a Saudi child: a case report.

The SLC29A3 variant, neutrophilic dermatosis, and hyperferritinemia imitate systemic juvenile idiopathic arthritis in a Saudi child: a case report.

The SLC29A3 variant, neutrophilic dermatosis, and hyperferritinemia imitate systemic juvenile idiopathic arthritis in a Saudi child: a case report.

Genetic defects of SLC29A3 result in a wide range of syndromic histiocytosis that encompasses H syndrome. Patients with SLC29A3 variants typically have hyperpigmentation, hypertrichosis, hepatosplenomegaly, sensorineural hearing loss, diabetes mellitus, and hypogonadism. Herein, we identify a novel phenotype in a girl presenting with clinical and laboratory findings similar to systemic juvenile arthritis and hyperferritinemia. Exome sequencing identified a homozygous variant in SLC29A3 (NM_018344.5: c.707C>T [p.T236M]). Our patient did not show the cardinal features of the broad spectrum of SLC29A3-related disorders. She demonstrated remarkable improvement in her clinical and laboratory manifestations after starting interleukin-1 blockade (Anakinra). Recent research suggests that SLC29A3-related disorders are accompanied with autoinflammation and autoimmunity due to an overactive inflammasome pathway, which is most likely induced by mitochondrial and lysosomal dysfunction. Hence, our findings may expand the phenotypic features of the SLC29A3 variant. Patients with the SLC29A3 variant and systemic inflammation may benefit from interleukin-1 blockade as a therapeutic option.

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来源期刊
CiteScore
2.30
自引率
5.00%
发文量
39
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