Makwana Rikhil, Patel Rahi, O'Neill Rosemary, Marchi Elaine, Lyon Gholson J
Department of Human Genetics, New York State Institute for Basic Research in Developmental Disabilities, Staten Island, NY, USA.
George A. Jervis Clinic, New York State Institute for Basic Research in Developmental Disabilities, Staten Island, NY, USA.
Pediatr Cardiol. 2025 Apr 28. doi: 10.1007/s00246-025-03877-7.
The NatA complex is composed of the NAA10, NAA15, and HYPK sub-units. It is primarily responsible for N-terminal acetylation, a critical post-translational modification in eukaryotes. Pathogenic variants within NAA10 cause Ogden Syndrome (OS), which is characterized by varying degrees of intellectual disability, hypotonia, developmental delay, and cardiac abnormalities. Although the cardiac manifestations of the disease have been described extensively in case reports, there has not been a study focusing on the cardiac manifestations and their recommended clinical cardiac management. In this study, we describe the cardiac manifestations of OS in a cohort of 85 probands. We found increased incidence of structural and electrophysiologic abnormalities, with particularly high prevalence of QT interval prolongation. Sub-analysis showed that male probands and those with variants within the NAA15-binding domain had more severe phenotypes than females or those with variants outside of the NAA15-binding domain. Our results suggest that an OS diagnosis should be accompanied by full cardiac workup with emphasis on echocardiogram for structural defects and EKG/Holter monitoring for electrophysiologic abnormalities. Additionally, we strongly recommend that the use of QT-prolonging drugs be followed up with routine electrophysiological monitoring or consultation with a pediatric cardiologist. We hope this study guides clinicians and caregivers treating patients with OS and moves the field toward a standardized diagnostic workup for patients with this condition.
NatA复合物由NAA10、NAA15和HYPK亚基组成。它主要负责N端乙酰化,这是真核生物中一种关键的翻译后修饰。NAA10内的致病变异会导致奥格登综合征(OS),其特征为不同程度的智力残疾、肌张力减退、发育迟缓以及心脏异常。尽管该疾病的心脏表现已在病例报告中得到广泛描述,但尚未有研究聚焦于心脏表现及其推荐的临床心脏管理。在本研究中,我们描述了85名先证者队列中OS的心脏表现。我们发现结构和电生理异常的发生率增加,尤其是QT间期延长的患病率很高。亚分析表明,男性先证者以及NAA15结合域内有变异的先证者比女性或NAA15结合域外有变异的先证者具有更严重的表型。我们的结果表明,OS诊断应伴有全面的心脏检查,重点是通过超声心动图检查结构缺陷,通过心电图/动态心电图监测电生理异常。此外,我们强烈建议对使用延长QT间期药物的患者进行常规电生理监测或咨询儿科心脏病专家。我们希望这项研究能指导治疗OS患者的临床医生和护理人员,并推动该领域朝着对这种疾病患者进行标准化诊断检查的方向发展。