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巴勒斯坦IPEX综合征病例报告:详细的家族鉴定及具有相同缺陷的疾病范围

A case report of IPEX syndrome in Palestine: detailed family identification and breadth of disorders with the same defect.

作者信息

Malhis Lana, AbdalSalam Zeidan, Njoum Yumna, Abdelhaq Anan, Sharaf Muna

机构信息

Faculty of Medicine, Najah National University, Nablus, Palestine.

Faculty of Medicine, Al-Quds University, Jerusalem, Palestine.

出版信息

Front Pediatr. 2024 Sep 20;12:1438816. doi: 10.3389/fped.2024.1438816. eCollection 2024.

Abstract

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.

摘要

免疫失调、多内分泌腺病、肠病、X连锁(IPEX)综合征是一种单基因疾病,其特征是由于编码叉头框P3(FOXP3)转录因子的基因功能丧失突变导致多系统自身免疫,FOXP3转录因子对CD4 +调节性T(T-reg)细胞的发育、成熟和维持很重要。全球已确诊的患者不到300例。IPEX的发病率低于1:1,000,000。在此,我们报告一例15日龄男性病例,该患儿出生后15天因呼吸窘迫入住新生儿重症监护病房(NICU)。他被发现因糖尿病酮症酸中毒(DKA)导致代谢性酸中毒。在NICU住院期间,他出现惊厥并插管一个月。他被诊断为新生儿糖尿病。他还反复出现呼吸道感染、多次腹泻皮疹发作和脑膜炎。7个月大时,基因检测确诊为携带FOXP3突变的IPEX,具体为FOXP3基因的p.(Pro75Leu)变异。随后,多名家庭成员被确诊。同一家庭中的个体尽管携带相同突变,但在器官受累和发病时间上观察到独特的变异性,这是一个显著特点,特别是考虑到FOXP3基因在男性中的单等位基因表达。这种现象强烈表明存在修饰基因,它们在IPEX综合征的发病机制中起重要作用。该病例报告强调了在新生儿糖尿病病例中临床怀疑IPEX的重要性。它还突出了儿科患者中罕见遗传病管理的挑战。它还强调IPEX基因型具有广泛的表型。该病例被认为是巴勒斯坦有记录的首例IPEX病例。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/0d5e/11449736/20eff58ae07b/fped-12-1438816-g001.jpg

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