Tilton Richard K, Wilkens Alisha, Krantz Ian D, Izumi Kosuke
Division of Human Genetics, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania; Temple University School of Medicine, Philadelphia, Pennsylvania.
Am J Med Genet A. 2014 May;164A(5):1130-5. doi: 10.1002/ajmg.a.36413. Epub 2014 Feb 6.
Pallister-Killian syndrome (PKS) is a sporadic multisystem genetic diagnosis characterized by facial dysmorphia, variable developmental delay and intellectual impairment, hypotonia, hearing loss, seizures, differences in skin pigmentation, temporal alopecia, diaphragmatic hernia, congenital heart defects, and other systemic abnormalities. Although congenital heart defects have been described in association with PKS, the full spectrum of heart disease is still not entirely known. Here, we describe the pattern of cardiac findings of 81 probands with PKS who have had at least one cardiac evaluation, demonstrating structural heart difference in 37% of our cohort (n = 30). Septal defects such as atrial or ventricular septal defects (n = 12) were the most commonly seen congenital heart differences. Additional findings included the occasional occurrence of bicuspid aortic valve, aortic dilatation, and cardiac hypertrophy/cardiomyopathy. We suggest cardiac evaluation for all individuals with PKS at the time of diagnosis as well as subsequent longitudinal follow-up to monitor for the development of cardiomyopathy and aortic dilatation.
帕利斯特-基利安综合征(PKS)是一种散发性多系统基因诊断疾病,其特征包括面部畸形、不同程度的发育迟缓与智力障碍、肌张力减退、听力丧失、癫痫发作、皮肤色素沉着差异、颞部脱发、膈疝、先天性心脏缺陷及其他全身异常。虽然先天性心脏缺陷已被描述与PKS相关,但心脏病的全貌仍不完全清楚。在此,我们描述了81例至少接受过一次心脏评估的PKS先证者的心脏检查结果模式,显示我们队列中有37%(n = 30)存在结构性心脏差异。诸如房间隔或室间隔缺损(n = 12)等间隔缺损是最常见的先天性心脏差异。其他发现包括偶尔出现的二叶主动脉瓣、主动脉扩张以及心脏肥大/心肌病。我们建议在诊断时对所有PKS患者进行心脏评估,并在随后进行长期随访,以监测心肌病和主动脉扩张的发展。