Pagani Riccardo, Lorioli Laura, Favini Francesca, Severi Eleonora, Salvi Marco, Pezzani Lidia, Iascone Maria, Migliazza Lucia, Pellegrinelli Claudia, Cheli Maurizio, Provenzi Massimo, Mangili Giovanna
Department of Pediatrics, Milano-Bicocca University, Fondazione IRCCS Ospedale San Gerardo dei Tintori, Monza, Italy.
Department of Neonatology, ASST Ospedale Papa Giovanni XXIII, Bergamo, Italy.
Front Pediatr. 2025 Mar 7;13:1550643. doi: 10.3389/fped.2025.1550643. eCollection 2025.
Leukocyte Adhesion Defects (LADs) are a group of rare autosomal recessive immune disorders characterized by constitutional defects in the process of leukocyte migration. Among these, LAD-III is the rarest, with only a few cases documented in scientific literature. It is caused by mutations in the FERMT3 gene, impairing integrin function in both white blood cells and platelets. Thus, patients exhibit a variable degree of immunodeficiency along with a severe bleeding tendency referred to as "Glanzmann-like", due to dysfunctional platelet GPIIb/IIIa. The diagnosis of LAD-III is typically made in infancy or early childhood, following medical evaluations for recurrent infections and bleeding episodes. Here we report the case of a female newborn admitted to our NICU at day four of life with a history of petechial rash and gross hematuria. Radiological and endoscopic assessments revealed a hemangioma-like lesion of the bladder wall. Blood exams showed persistent leukocytosis without signs of infection, associated with mild thrombocytopenia and normocytic anemia. Notably, platelet function assays demonstrated defective aggregation with all agonists tested. Next generation sequencing analysis identified a homozygous nonsense mutation in the FERMT3 gene, ensuring early access to hematopoietic stem cell transplantation, which is the only curative treatment. To the best of our knowledge, this is the first reported case of LAD-III diagnosed in the neonatal period and the first to associate this rare disorder with bladder angiomatosis. This case highlights the importance of early genetic evaluations in newborns with unexplained hematological abnormalities and bleeding tendencies.
白细胞黏附缺陷(LADs)是一组罕见的常染色体隐性免疫疾病,其特征是白细胞迁移过程中存在先天性缺陷。其中,LAD-III最为罕见,科学文献中仅记载了少数病例。它由FERMT3基因突变引起,损害白细胞和血小板中的整合素功能。因此,患者表现出不同程度的免疫缺陷,同时由于血小板糖蛋白IIb/IIIa功能失调,还存在严重的出血倾向,称为“类Glanzmann样”。LAD-III的诊断通常在婴儿期或幼儿期做出,是在对反复感染和出血发作进行医学评估之后。在此,我们报告一例女性新生儿,出生后第四天因瘀点皮疹和肉眼血尿病史入住我们的新生儿重症监护病房(NICU)。放射学和内镜评估显示膀胱壁有血管瘤样病变。血液检查显示持续白细胞增多但无感染迹象,伴有轻度血小板减少和正细胞性贫血。值得注意的是,血小板功能检测显示对所有测试的激动剂的聚集均有缺陷。下一代测序分析在FERMT3基因中鉴定出一个纯合无义突变,确保了早期获得造血干细胞移植,这是唯一的治愈性治疗方法。据我们所知,这是首例在新生儿期诊断出的LAD-III病例,也是首例将这种罕见疾病与膀胱血管瘤病相关联的病例。该病例突出了对有不明血液学异常和出血倾向的新生儿进行早期基因评估的重要性。