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,Évelin Aline Zanardo,A. Rangel‐Santos,Gil Monteiro Novo‐Filho,Gleyson Francisco,Lucas Liro,A. M. Nascimento,Samar N. 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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