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。

IF 2.1 3区 医学 Q2 PEDIATRICS Frontiers in Pediatrics Pub Date : 2024-09-20 eCollection Date: 2024-01-01 DOI:10.3389/fped.2024.1438816
Lana Malhis, Zeidan AbdalSalam, Yumna Njoum, Anan Abdelhaq, Muna Sharaf
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引用次数: 0

摘要

免疫失调、多内分泌病、肠病、X-连锁(IPEX)综合征是一种单基因遗传性疾病,其特征是由于编码叉头盒 P3(FOXP3)转录因子的基因发生功能缺失突变而继发的多系统自身免疫性疾病,该转录因子对 CD4 + 调节性 T(T-调节)细胞的发育、成熟和维持非常重要。全世界已发现的患者不到 300 人。IPEX 的发病率低于 1:1,000,000。在此,我们介绍一例 15 天大的男婴,他在分娩后 15 天因呼吸窘迫被送入新生儿重症监护室。他被发现因 DKA 导致代谢性酸中毒。在新生儿重症监护室住院期间,他经历了癫痫发作,并插管一个月。他被诊断患有新生儿糖尿病。他还经历了反复呼吸道感染、多次腹泻皮疹和脑膜炎。7 个月大时,基因检测证实 IPEX 患有 FOXP3 基因突变,特别是 FOXP3 基因的 p.(Pro75Leu) 变异。随后,又有多名家庭成员被确诊。尽管携带相同的基因突变,但在同一家族中,不同个体的器官受累情况和发病时间存在独特的差异,这是一个与众不同的方面,特别是考虑到 FOXP3 基因在男性中的单倍表达。这一现象有力地表明,存在着在 IPEX 综合征发病机制中起重要作用的修饰基因。本病例强调了临床怀疑新生儿 DM 病例中 IPEX 的重要性。该病例还强调了儿科罕见遗传性疾病管理方面的挑战。它还强调了 IPEX 基因型具有广泛的表型。本病例被认为是巴勒斯坦首例有记录的 IPEX 病例。
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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.

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来源期刊
Frontiers in Pediatrics
Frontiers in Pediatrics Medicine-Pediatrics, Perinatology and Child Health
CiteScore
3.60
自引率
7.70%
发文量
2132
审稿时长
14 weeks
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.
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