Panizo Morgado Elena, Darnaude María Teresa, Torres Mohedas Julián, Benedit María, Cervera Bravo Áurea
Departments of Pediatrics.
Genetics, Hospital Universitario de Móstoles.
J Pediatr Hematol Oncol. 2020 Oct;42(7):e686-e688. doi: 10.1097/MPH.0000000000001742.
Hereditary spherocytosis arises from alterations in the genes encoding red blood cell membrane proteins. Although its diagnosis is mostly clinical, recent advances in next-generation sequencing (NGS) technologies have allowed for a faster cost-effective gene-based diagnosis. We report the case of a boy with spherocytic anemia and development delay in whom a de novo 2.84-Mb deletion at chromosome 14 including SPTB (ß-spectrin gene) was identified by array-based comparative genomic hybridization. This alteration, consistent with de novo spherocytosis, was missed by a NGS gene panel. When associated with other symptoms, especially neurologic, NGS may not be appropriate to genetically diagnose spherocytic anemia.
遗传性球形红细胞增多症源于编码红细胞膜蛋白的基因发生改变。尽管其诊断主要依靠临床症状,但新一代测序(NGS)技术的最新进展使得基于基因的诊断能够更快且更具成本效益。我们报告了一例患有球形红细胞贫血和发育迟缓的男孩病例,通过基于阵列的比较基因组杂交技术,在其14号染色体上发现了一个包括SPTB(β-血影蛋白基因)在内的2.84 Mb的新发缺失。这种改变与新发球形红细胞增多症相符,但被NGS基因检测 panel遗漏了。当与其他症状尤其是神经系统症状相关联时,NGS可能不适用于遗传性球形红细胞贫血的基因诊断。