Fonseca Madalena, Abrantes Francisco, Pinho Sara, Esteves Isabel, Salgado Catarina, Gonçalves Carolina, Ferrão Anabela
Pediatrics, Hospital de Santa Maria, Unidade Local de Saúde de Santa Maria, Lisbon, PRT.
Pediatrics and Genetics, Hospital de Santa Maria, Unidade Local de Saúde de Santa Maria, Lisbon, PRT.
Cureus. 2025 Jun 22;17(6):e86531. doi: 10.7759/cureus.86531. eCollection 2025 Jun.
Phosphoglucomutase 3 (PGM3) deficiency (OMIM (Online Mendelian Inheritance in Man) #615816) is a rare autosomal recessive congenital disorder of glycosylation that disrupts multiple glycosylation pathways, with few cases reported in the literature. It leads to a broad clinical spectrum ranging from hyper-IgE syndrome (HIES)-like features to severe combined immunodeficiency (SCID). We report a case of a 17-year-old female of Brazilian origin, referred to our center in Portugal for investigation of persistent neutropenia. Her medical history included recurrent infections in early childhood, severe eczema, and autism spectrum disorder. She exhibited persistent neutropenia and T-cell lymphopenia, with elevated IgE levels. Genetic analysis using a next-generation sequencing panel for primary immunodeficiencies identified compound heterozygous likely pathogenic variants in PGM3: a missense variant (c.1475C>T, p.(Thr492Ile)) and a complete gene deletion in the other allele, confirming the diagnosis of PGM3 deficiency. Chronic neutropenia was the main finding that prompted the genetic investigation. Although it is not a defining feature of PGM3 deficiency, it has been reported in nearly half of the cases. In this patient, the clinical presentation has been comparatively milder than the severe phenotypes described in the literature, which highlights the phenotypic variability of this condition and the need for clinical suspicion, even when classical features are absent. The genetic diagnosis has important implications for clinical follow-up and enables appropriate genetic counseling. This case illustrates the clinical variability of PGM3 deficiency and reinforces the role of genetic testing in clarifying the diagnosis, guiding management, and informing long-term follow-up in rare inborn errors of immunity.
磷酸葡萄糖变位酶3(PGM3)缺乏症(OMIM(《人类在线孟德尔遗传》)#615816)是一种罕见的常染色体隐性先天性糖基化障碍疾病,会扰乱多种糖基化途径,文献中报道的病例较少。它会导致广泛的临床症状,从高免疫球蛋白E综合征(HIES)样特征到严重联合免疫缺陷(SCID)。我们报告了一例17岁的巴西裔女性病例,她因持续性中性粒细胞减少症被转诊至我们位于葡萄牙的中心进行检查。她的病史包括幼儿期反复感染、严重湿疹和自闭症谱系障碍。她表现出持续性中性粒细胞减少症和T细胞淋巴细胞减少症,免疫球蛋白E水平升高。使用针对原发性免疫缺陷的下一代测序 panel 进行基因分析,在PGM3中鉴定出复合杂合的可能致病变异:一个错义变异(c.1475C>T,p.(Thr492Ile)),另一个等位基因存在完整基因缺失,从而确诊为PGM3缺乏症。慢性中性粒细胞减少症是促使进行基因调查的主要发现。虽然它不是PGM3缺乏症的决定性特征,但在近一半的病例中都有报道。在该患者中,临床表现相对比文献中描述的严重表型更为温和,这突出了这种疾病的表型变异性以及即使没有典型特征也需要临床怀疑的必要性。基因诊断对临床随访具有重要意义,并能够进行适当的遗传咨询。该病例说明了PGM3缺乏症的临床变异性,并强化了基因检测在明确诊断、指导管理以及为罕见的先天性免疫缺陷病的长期随访提供信息方面的作用。