Gonzalez Mercedes E, Votava Henry J, Lipkin George, Sanchez Miguel
Department of Dermatology, New York University, USA.
Dermatol Online J. 2009 Aug 15;15(8):17.
Pseudoxanthoma elasticum is an incurable, autosomal-recessive, genetic disorder that is caused by mutations in the ABCC6 gene. It is characterized by progressive mineralization and fragmentation of the elastic fibers in the skin, retina, and blood vessels. The characteristic cutaneous features bring the patient to medical attention, but morbidity is related to the degree of extracutaneous involvement. The disease is progressive with phenotypic variability and no definite genotype-phenotype correlation. Treatment is supportive and is directed at prevention and early detection of adverse ocular and cardiovascular sequelae. We present two siblings with pseudoxanthoma elasticum, who have considerable differences in disease related morbidity, which highlights intra-familiar phenotypic heterogeneity.
弹性假黄瘤是一种无法治愈的常染色体隐性遗传病,由ABCC6基因突变引起。其特征是皮肤、视网膜和血管中的弹性纤维进行性矿化和断裂。典型的皮肤特征引起患者就医,但发病率与皮肤外受累程度有关。该疾病呈进行性发展,具有表型变异性,且不存在明确的基因型-表型相关性。治疗以支持治疗为主,旨在预防和早期发现不良的眼部和心血管后遗症。我们报告了两名患有弹性假黄瘤的同胞,他们在疾病相关发病率上存在显著差异,这突出了家族内表型异质性。