Shahad Alansari, Alhanouf Alsaleem, Tariq Alzaid, Maad Galal, Noura Alyahya, Sulaiman M Al-Mayouf
{"title":"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。","authors":"Shahad Alansari, Alhanouf Alsaleem, Tariq Alzaid, Maad Galal, Noura Alyahya, Sulaiman M Al-Mayouf","doi":"10.4078/jrd.22.0054","DOIUrl":null,"url":null,"abstract":"<p><p>Genetic defects of <i>SLC29A3</i> result in a wide range of syndromic histiocytosis that encompasses H syndrome. Patients with <i>SLC29A3</i> 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 <i>SLC29A3</i> (NM_018344.5: c.707C>T [p.T236M]). Our patient did not show the cardinal features of the broad spectrum of <i>SLC29A3</i>-related disorders. She demonstrated remarkable improvement in her clinical and laboratory manifestations after starting interleukin-1 blockade (Anakinra). Recent research suggests that <i>SLC29A3</i>-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 <i>SLC29A3</i> variant. Patients with the <i>SLC29A3</i> variant and systemic inflammation may benefit from interleukin-1 blockade as a therapeutic option.</p>","PeriodicalId":56161,"journal":{"name":"Journal of Rheumatic Diseases","volume":"30 2","pages":"133-137"},"PeriodicalIF":2.2000,"publicationDate":"2023-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/cc/35/jrd-30-2-133.PMC10324931.pdf","citationCount":"1","resultStr":"{\"title\":\"The SLC29A3 variant, neutrophilic dermatosis, and hyperferritinemia imitate systemic juvenile idiopathic arthritis in a Saudi child: a case report.\",\"authors\":\"Shahad Alansari, Alhanouf Alsaleem, Tariq Alzaid, Maad Galal, Noura Alyahya, Sulaiman M Al-Mayouf\",\"doi\":\"10.4078/jrd.22.0054\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Genetic defects of <i>SLC29A3</i> result in a wide range of syndromic histiocytosis that encompasses H syndrome. Patients with <i>SLC29A3</i> 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 <i>SLC29A3</i> (NM_018344.5: c.707C>T [p.T236M]). Our patient did not show the cardinal features of the broad spectrum of <i>SLC29A3</i>-related disorders. She demonstrated remarkable improvement in her clinical and laboratory manifestations after starting interleukin-1 blockade (Anakinra). Recent research suggests that <i>SLC29A3</i>-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 <i>SLC29A3</i> variant. Patients with the <i>SLC29A3</i> variant and systemic inflammation may benefit from interleukin-1 blockade as a therapeutic option.</p>\",\"PeriodicalId\":56161,\"journal\":{\"name\":\"Journal of Rheumatic Diseases\",\"volume\":\"30 2\",\"pages\":\"133-137\"},\"PeriodicalIF\":2.2000,\"publicationDate\":\"2023-04-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/cc/35/jrd-30-2-133.PMC10324931.pdf\",\"citationCount\":\"1\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Rheumatic Diseases\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.4078/jrd.22.0054\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"RHEUMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Rheumatic Diseases","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.4078/jrd.22.0054","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"RHEUMATOLOGY","Score":null,"Total":0}
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.