The Section of Pediatric Cardiology, Texas Children's Hospital, Houston, Texas, USA.
Am J Med Genet A. 2011 Dec;155A(12):3030-4. doi: 10.1002/ajmg.a.33856. Epub 2011 Oct 18.
Coffin-Lowry syndrome (CLS) is an X-linked dominant condition characterized by moderate to severe mental retardation, characteristic facies, and hand and skeletal malformations. The syndrome is due to mutations in the gene that encodes the ribosomal protein S6 kinase-2, a growth factor-regulating protein kinase located on Xp22.2. Cardiac anomalies are known to be associated with CLS. Left ventricular noncompaction (LVNC) is a clinically heterogeneous disorder characterized by left ventricular (LV) myocardial trabeculations and intertrabecular recesses that communicate with the LV cavity. Patients may present with a variety of clinical phenotypes, ranging from a complete absence of symptoms to a rapid, progressive decline in LV systolic and diastolic function, resulting in congestive heart failure, malignant ventricular tachyarrhythmias, and systemic thromboembolic events. Restrictive cardiomyopathy is an uncommon primary cardiomyopathy characterized by biatrial enlargement, normal or decreased biventricular volume, impaired ventricular filling, and normal or near-normal systolic function. We describe a patient with CLS and LVNC with a restrictive pattern, as documented by echocardiography and cardiac catheterization. To our knowledge, there have been no previous reports of concomitant CLS and LVNC. On the basis of our case, we suggest that patients with CLS be screened not only for congenital structural heart defects but also for LVNC cardiomyopathy.
Coffin-Lowry 综合征(CLS)是一种 X 连锁显性疾病,其特征为中度至重度智力障碍、特征性面容以及手和骨骼畸形。该综合征是由于编码核糖体蛋白 S6 激酶-2 的基因突变引起的,S6 激酶-2 是一种位于 Xp22.2 的生长因子调节蛋白激酶。已知心脏异常与 CLS 相关。左心室心肌致密化不全(LVNC)是一种临床表现异质性的疾病,其特征为左心室(LV)心肌小梁和小梁间陷窝与 LV 腔相通。患者可能表现出多种临床表型,从完全无症状到 LV 收缩和舒张功能迅速进行性下降,导致充血性心力衰竭、恶性室性心律失常和全身血栓栓塞事件。限制型心肌病是一种不常见的原发性心肌病,其特征为双心房增大、双心室容积正常或减小、心室充盈受损以及收缩功能正常或接近正常。我们描述了一例 CLS 合并 LVNC 的患者,其表现为限制型模式,通过超声心动图和心导管检查得到证实。据我们所知,以前没有报道过同时存在 CLS 和 LVNC。基于我们的病例,我们建议对 CLS 患者进行筛查,不仅要筛查先天性结构性心脏缺陷,还要筛查 LVNC 心肌病。