医学
表型
遗传学
微缺失综合征
临床表型
基因
生物
作者
Marília M. Montenegro,Débora Camilotti,Caio Robledo D’Anglioli Costa Quaio,Yanca Gasparini Oliveira,Évelin Aline Zanardo,Andréia Rangel-Santos,Gil Monteiro Novo‐Filho,Gleyson Francisco,Lucas Liro,A. Nascimento,Samar Nasser Chehimi,Diogo Cordeiro de Queiroz Soares,Ana Cristina Victorino Krepischi,Marcília Sierro Grassi,Rachel Sayuri Honjo,Patrícia Palmeira,Chong Ae Kim,Magda Carneiro‐Sampaio,Carla Rosenberg,Leslie Domenici Kulikowski
标识
DOI:10.1016/j.jpeds.2022.08.051
摘要
To report the effectiveness of early molecular diagnosis in the clinical management of rare diseases, presenting 8 patients with 8p23.1DS who have clinical features that overlap the phenotypic spectrum of 22q11.2DS.This report is part of a previous study that aims to provide a precocious molecular diagnosis of the 22q11.2 deletion syndrome in 118 infants with congenital heart disease. To confirm the clinical diagnosis, patients underwent comparative genomic screening by the multiplex ligation-dependent probe amplification (MLPA) assay with the SALSA MLPA probemix kits P064-B2, P036-E1, P070-B2, P356-A1, and P250- B1. Subsequently, the patients performed the genomic microarray using the Infinium CytoSNP-850K BeadChip to confirm the deletion, determine the breakpoints of the deletion, and search for genomic copy number variations.MLPA performed with 3 different kits revealed the 8p23.1 typical deletion involving the PPP1R3B, MSRA, and GATA4 genes in the 5 patients. The array analysis was performed on these 5 patients and 3 other patients (8 patients) who also had clinical suspicion of 22q11 deletion (8 patients) allowed a precise definition of the breakpoints and excluded other genomic abnormalities.Cytogenomic screening was efficient in establishing a differential diagnosis and ruling out the presence of other concomitant syndromes. The clinical picture of the 8p23.1 deletion syndrome is challenging; however, cytogenomic tools can provide an exact diagnosis and help to clarify the genotype-phenotype complexity of these patients. Our reports underline the importance of early diagnosis and clinical follow-up of microdeletion syndromes.
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