Aydin Hilal, Esener Zeynep, Bolat Hilmi, Aytaç Adil
Balikesir University, Faculty of Medicine, Department of Pediatrics, Balikesir, Türkiye.
Balikesir University, Faculty of Medicine, Department of Medical Genetics, Balikesir, Türkiye.
Int J Dev Neurosci. 2025 Aug;85(5):e70039. doi: 10.1002/jdn.70039.
Glucose transporter type 1 deficiency syndrome (GLUT1DS) affects all age groups, from infants to adolescents, and involves age-specific symptoms. Nonclassic GLUT1 DS is observed in 10% of cases, in which seizures are not observed, and the condition involves a milder accompanying phenotype and paroxysmal dyskinesias. Cranial imaging findings in cases of GLUT1 DS are variable. The purpose of this report is to describe rare genetic variants in two cases of GLUT1 DS with cortical dysplasia detected at magnetic resonance imaging (MRI) and exhibiting differing clinical presentations and to discuss the relationship between them. Two cases presenting to the Balıkesir University Medical Faculty paediatric neurology clinic, Türkiye, between 01.08.2019 and 01.12.2024 due to seizures and inability to speak/numbness in the hands and arms, diagnosed as GLUT1 DS, and with cortical dysplasia, were included. The patients' files, MRI and physical examination findings and family pedigrees were evaluated. We detected two different pathogenic and likely pathogenic variants in SLC2A1 (NM_006516.3) in patients from unrelated families. Patient 1 exhibited a heterozygous c. 1208C > T variant and patient 2 a heterozygous likely c. 278G > A variant. In conclusion, the careful evaluation of patients with structural brain damage and determination of the molecular aetiology of underlying inherited metabolic diseases are highly important in terms of the provision of treatment, prognosis, and genetic counselling. Although cortical malformations have been reported in patients with GLUT1 DS, the mechanism involved remains unclear, and this report highlights the potential relationship between cortical dysplasia and specific genotypes in GLUT1 DS. Further prospective observational and functional studies involving larger numbers of cases and centres are now needed.
1型葡萄糖转运体缺乏综合征(GLUT1DS)影响从婴儿到青少年的所有年龄组,并涉及特定年龄的症状。10%的病例为非典型GLUT1DS,这些病例未出现癫痫发作,病情伴有较轻微的表型和阵发性运动障碍。GLUT1DS病例的头颅影像学表现各异。本报告的目的是描述两例GLUT1DS患者中罕见的基因变异,这两例患者在磁共振成像(MRI)检查时发现有皮质发育异常,临床表现不同,并探讨它们之间的关系。纳入了2019年8月1日至2024年12月1日期间因癫痫发作以及手部和手臂无法说话/麻木而就诊于土耳其巴勒克埃西尔大学医学院儿科神经科诊所、被诊断为GLUT1DS且伴有皮质发育异常的两例患者。对患者的病历、MRI和体格检查结果以及家族谱系进行了评估。我们在来自无关家族的患者中检测到SLC2A1(NM_006516.3)基因的两种不同的致病和可能致病变异。患者1表现为杂合的c.1208C>T变异,患者2表现为杂合的可能的c.278G>A变异。总之,对于有结构性脑损伤的患者进行仔细评估并确定潜在遗传性代谢疾病的分子病因,在治疗、预后和遗传咨询方面非常重要。虽然GLUT1DS患者中曾有皮质畸形的报道,但其涉及的机制仍不清楚,本报告强调了GLUT1DS中皮质发育异常与特定基因型之间的潜在关系。现在需要涉及更多病例和中心的进一步前瞻性观察和功能研究。