Duz Mehmet B, Kirat Emre, Coucke Paul J, Koparir Erkan, Gezdirici Alper, Paepe Anne De, Callewaert Bert, Seven Mehmet
aDepartment of Medical Genetics, Cerrahpasa Medical School, Istanbul University, Istanbul, Turkey bCenter for Medical Genetics, Ghent University Hospital, Ghent, Belgium.
Clin Dysmorphol. 2017 Jul;26(3):142-147. doi: 10.1097/MCD.0000000000000179.
Autosomal dominant cutis laxa (ADCL, OMIM #123700) is a rare connective tissue disorder characterized by loose, redundant skin folds that may be apparent form birth or appear later in life. Most severely affected areas are the neck, axillar regions, trunk, and groin. Typically, patients present with characteristic facial features including a premature aged appearance, long philtrum, a high forehead, large ears, and a beaked nose. Cardiovascular and pulmonary complications include bicuspid aortic valves, aortic root dilatation, and emphysema. Sporadically, these complications have been documented to cause premature death. Several rare findings including urogenital anomalies and gastroesophageal problems can be also occur. Most patients harbor a frameshift mutation in one of the five last exons of the ELN gene (ADCL1, OMIM #123700), whereas one patient was described to have a tandem duplication in the FBLN5 gene (ADCL2, OMIM #614434). Here, we present a female ADCL patient, from a consanguineous family, with a novel mutation in ELN and review 39 previously reported ADCL patients. All patients have various skin findings, whereas cardiovascular, pulmonary findings, and multiple hernia were present in 61, 28, and 38% of patients, respectively. Strabismus, urogenital anomalies, gastroesophageal problems, and scoliosis may rarely be present. A clear definition of the ADCL syndrome can enable more accurate genetic counseling.
常染色体显性遗传性皮肤松弛症(ADCL,OMIM编号#123700)是一种罕见的结缔组织疾病,其特征为皮肤褶皱松弛、多余,出生时即可出现,也可能在日后出现。受影响最严重的部位是颈部、腋窝、躯干和腹股沟。通常,患者具有特征性的面部特征,包括早衰外观、长人中、高额、大耳和钩状鼻。心血管和肺部并发症包括二尖瓣主动脉瓣、主动脉根部扩张和肺气肿。偶有记录显示,这些并发症会导致过早死亡。还可能出现一些罕见的症状,包括泌尿生殖系统异常和胃食管问题。大多数患者在ELN基因(ADCL1,OMIM编号#123700)最后五个外显子之一中存在移码突变,而有一名患者被描述为FBLN5基因存在串联重复(ADCL2,OMIM编号#614434)。在此,我们报告一名来自近亲家庭的女性ADCL患者,其ELN基因存在新的突变,并回顾39例先前报道的ADCL患者。所有患者均有各种皮肤表现,而心血管、肺部表现及多发疝分别见于61%、28%和38%的患者。斜视、泌尿生殖系统异常、胃食管问题和脊柱侧弯可能很少出现。对ADCL综合征的明确定义有助于进行更准确的遗传咨询。