From the General Paediatrics and Infectious and Tropical Diseases Department, Hospital La Paz-IdiPAZ, Madrid, Spain.
Pediatric Infectious Diseases and Immunodeficiencies Unit.
Pediatr Infect Dis J. 2019 Feb;38(2):157-160. doi: 10.1097/INF.0000000000002058.
Newborn screening for severe combined immunodeficiency using T-cell receptor excision circles allows prompt diagnosis and initiation of supportive and curative therapy thereby reducing morbidity and mortality. However, profound combined immunodeficiencies with normal numbers of nonfunctional T cells will go undetected. We present a patient with calcium release-activated calcium channel gene (ORAI1) deficiency and normal T-cell receptor excision circle numbers observed after diagnosis at the age of 14 months who suffered from disseminated fatal cytomegalovirus and Pneumocystis jirovecii infection, demonstrating a potential pitfall of the current newborn screening program.
采用 T 细胞受体切除环进行新生儿严重联合免疫缺陷筛查可及时诊断,并开始支持和治疗,从而降低发病率和死亡率。然而,具有正常数量无功能 T 细胞的严重联合免疫缺陷则无法被检测到。我们报告了一例钙释放激活钙通道基因(ORAI1)缺陷的患者,该患者在 14 个月大时被诊断,当时 T 细胞受体切除环的数量正常,他随后发生了播散性致命性巨细胞病毒和卡氏肺孢子虫感染,这表明现行新生儿筛查方案存在潜在缺陷。