Milone Roberta, Cesario Claudia, Goldoni Marina, Pasquariello Rosa, Fusilli Caterina, Giovannetti Agnese, Giglio Sabrina, Novelli Antonio, Caputo Viviana, Cioni Giovanni, Mazza Tommaso, Battaglia Agatino, Bernardini Laura, Battini Roberta
Department of Developmental Neuroscience, IRCCS Stella Maris Foundation, Pisa, Italy.
Laboratory of Medical Genetics, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
J Pediatr Genet. 2020 Sep 18;10(4):292-299. doi: 10.1055/s-0040-1716398. eCollection 2021 Dec.
The aim of this study was to evaluate the relationship between neurodevelopmental disorders, brain anomalies, and copy number variations (CNVs) and to estimate the diagnostic potential of cytogenomical microarray analysis (CMA) in individuals neuroradiologically characterized with intellectual developmental disorders (IDDs) isolated or associated with autism spectrum disorders (ASDs) and epilepsy (EPI), all of which were identified as a "synaptopathies." We selected patients who received CMA and brain magnetic resonance imaging (MRI) over a 7-year period. We divided them into four subgroups: IDD, IDD + ASD, IDD + EPI, and IDD + ASD + EPI. The diagnostic threshold of CMA was 16%. The lowest detection rate for both CMA and brain anomalies was found in IDD + ASD, while MRI was significantly higher in IDD and IDD + EPI subgroups. CMA detection rate was significantly higher in patients with brain anomalies, so CMA may be even more appropriate in patients with pathological MRI, increasing the diagnostic value of the test. Conversely, positive CMA in IDD patients should require an MRI assessment, which is more often associated with brain anomalies. Posterior fossa anomalies, both isolated and associated with other brain anomalies, showed a significantly higher rate of CMA positive results and of pathogenic CNVs. In the next-generation sequencing era, our study confirms once again the relevant diagnostic output of CMA in patients with IDD, either isolated or associated with other comorbidities. Since more than half of the patients presented brain anomalies in this study, we propose that neuroimaging should be performed in such cases, particularly in the presence of genomic imbalances.
本研究的目的是评估神经发育障碍、脑异常与拷贝数变异(CNV)之间的关系,并估计细胞基因组微阵列分析(CMA)对经神经放射学诊断为孤立性智力发育障碍(IDD)或合并自闭症谱系障碍(ASD)和癫痫(EPI)的个体的诊断潜力,所有这些均被视为“突触病”。我们选取了在7年期间接受CMA和脑磁共振成像(MRI)检查的患者。我们将他们分为四个亚组:IDD、IDD + ASD、IDD + EPI和IDD + ASD + EPI。CMA的诊断阈值为16%。CMA和脑异常的最低检出率均在IDD + ASD组中发现,而MRI在IDD组和IDD + EPI亚组中的检出率显著更高。脑异常患者的CMA检出率显著更高,因此CMA可能在MRI有病理表现的患者中更为适用,从而提高检查的诊断价值。相反,IDD患者CMA结果为阳性时应进行MRI评估,因为MRI更常与脑异常相关。孤立的或与其他脑异常相关的后颅窝异常,CMA阳性结果和致病性CNV的发生率均显著更高。在下一代测序时代,我们的研究再次证实了CMA对孤立性或合并其他疾病的IDD患者具有重要的诊断价值。由于本研究中超过一半的患者存在脑异常,我们建议在这种情况下应进行神经影像学检查,尤其是在存在基因组失衡的情况下。