Lana Malhis, Zeidan AbdalSalam, Yumna Njoum, Anan Abdelhaq, Muna Sharaf
{"title":"巴勒斯坦 IPEX 综合征病例报告:详细的家族鉴定和具有相同缺陷的疾病的广泛性。","authors":"Lana Malhis, Zeidan AbdalSalam, Yumna Njoum, Anan Abdelhaq, Muna Sharaf","doi":"10.3389/fped.2024.1438816","DOIUrl":null,"url":null,"abstract":"<p><p>Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is a monogenic disorder characterized by multi-systemic autoimmunity secondary to loss-of-function mutations in the gene coding the forkhead box P3 (FOXP3) transcription factor which is important for the development, maturation, and maintenance of CD4 + regulatory T (T-reg) cells. Fewer than 300 affected individuals have been identified worldwide. The occurrence of IPEX is below 1:1,000,000. Herein we present a case of a 15-day-old male who was admitted to NICU 15 days after delivery due to respiratory distress. He was found to have metabolic acidosis due to DKA. During his stay in the NICU, he experienced seizures and was intubated for a month. He was diagnosed with neonatal diabetes. He also experienced recurrent respiratory infections and multiple episodes of diarrhea rash, and meningitis. At the age of 7 months, genetic testing confirmed IPEX with FOXP3 mutation, specifically the p.(Pro75Leu) variant of the FOXP3 gene. Subsequently, multiple family members were diagnosed. The unique variability observed in organ involvement and presentation timing among individuals within the same family, despite carrying an identical mutation, is a distinctive aspect, particularly considering the monoallelic expression of the FOXP3 gene in males. This phenomenon strongly suggests the presence of modifying genes that play a significant role in the pathogenesis of IPEX syndrome. The case presentation underscores the importance of clinical suspicion of IPEX in cases of neonatal DM. It also highlights the challenges associated with managing rare genetic disorders in pediatric patients. It also emphasizes that the IPEX genotype has a wide phenotype. This case is considered the first documented case of IPEX in Palestine.</p>","PeriodicalId":12637,"journal":{"name":"Frontiers in Pediatrics","volume":null,"pages":null},"PeriodicalIF":2.1000,"publicationDate":"2024-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11449736/pdf/","citationCount":"0","resultStr":"{\"title\":\"A case report of IPEX syndrome in Palestine: detailed family identification and breadth of disorders with the same defect.\",\"authors\":\"Lana Malhis, Zeidan AbdalSalam, Yumna Njoum, Anan Abdelhaq, Muna Sharaf\",\"doi\":\"10.3389/fped.2024.1438816\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is a monogenic disorder characterized by multi-systemic autoimmunity secondary to loss-of-function mutations in the gene coding the forkhead box P3 (FOXP3) transcription factor which is important for the development, maturation, and maintenance of CD4 + regulatory T (T-reg) cells. Fewer than 300 affected individuals have been identified worldwide. The occurrence of IPEX is below 1:1,000,000. Herein we present a case of a 15-day-old male who was admitted to NICU 15 days after delivery due to respiratory distress. He was found to have metabolic acidosis due to DKA. During his stay in the NICU, he experienced seizures and was intubated for a month. He was diagnosed with neonatal diabetes. He also experienced recurrent respiratory infections and multiple episodes of diarrhea rash, and meningitis. At the age of 7 months, genetic testing confirmed IPEX with FOXP3 mutation, specifically the p.(Pro75Leu) variant of the FOXP3 gene. Subsequently, multiple family members were diagnosed. The unique variability observed in organ involvement and presentation timing among individuals within the same family, despite carrying an identical mutation, is a distinctive aspect, particularly considering the monoallelic expression of the FOXP3 gene in males. This phenomenon strongly suggests the presence of modifying genes that play a significant role in the pathogenesis of IPEX syndrome. The case presentation underscores the importance of clinical suspicion of IPEX in cases of neonatal DM. It also highlights the challenges associated with managing rare genetic disorders in pediatric patients. It also emphasizes that the IPEX genotype has a wide phenotype. 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A case report of IPEX syndrome in Palestine: detailed family identification and breadth of disorders with the same defect.
Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is a monogenic disorder characterized by multi-systemic autoimmunity secondary to loss-of-function mutations in the gene coding the forkhead box P3 (FOXP3) transcription factor which is important for the development, maturation, and maintenance of CD4 + regulatory T (T-reg) cells. Fewer than 300 affected individuals have been identified worldwide. The occurrence of IPEX is below 1:1,000,000. Herein we present a case of a 15-day-old male who was admitted to NICU 15 days after delivery due to respiratory distress. He was found to have metabolic acidosis due to DKA. During his stay in the NICU, he experienced seizures and was intubated for a month. He was diagnosed with neonatal diabetes. He also experienced recurrent respiratory infections and multiple episodes of diarrhea rash, and meningitis. At the age of 7 months, genetic testing confirmed IPEX with FOXP3 mutation, specifically the p.(Pro75Leu) variant of the FOXP3 gene. Subsequently, multiple family members were diagnosed. The unique variability observed in organ involvement and presentation timing among individuals within the same family, despite carrying an identical mutation, is a distinctive aspect, particularly considering the monoallelic expression of the FOXP3 gene in males. This phenomenon strongly suggests the presence of modifying genes that play a significant role in the pathogenesis of IPEX syndrome. The case presentation underscores the importance of clinical suspicion of IPEX in cases of neonatal DM. It also highlights the challenges associated with managing rare genetic disorders in pediatric patients. It also emphasizes that the IPEX genotype has a wide phenotype. This case is considered the first documented case of IPEX in Palestine.
期刊介绍:
Frontiers in Pediatrics (Impact Factor 2.33) publishes rigorously peer-reviewed research broadly across the field, from basic to clinical research that meets ongoing challenges in pediatric patient care and child health. Field Chief Editors Arjan Te Pas at Leiden University and Michael L. Moritz at the Children''s Hospital of Pittsburgh are supported by an outstanding Editorial Board of international experts. This multidisciplinary open-access journal is at the forefront of disseminating and communicating scientific knowledge and impactful discoveries to researchers, academics, clinicians and the public worldwide.
Frontiers in Pediatrics also features Research Topics, Frontiers special theme-focused issues managed by Guest Associate Editors, addressing important areas in pediatrics. In this fashion, Frontiers serves as an outlet to publish the broadest aspects of pediatrics in both basic and clinical research, including high-quality reviews, case reports, editorials and commentaries related to all aspects of pediatrics.