Al-Haggar Mohammad, Bakr Ashraf, Wahba Yahya, Coucke Paul J, El-Hussini Fatma, Hafez Mona, Eid Riham, Eid Abdel-Rahman, Sarhan Amr, Shaltout Ali, Hammad Ayman, Yahia Sohier, El-Rifaie Ahmad, Abdel-Hadi Dina
Department of Pediatrics, Genetics Unit, Faculty of Medicine, Mansoura University, Mansoura, Egypt.
Nephrology Unit, Faculty of Medicine, Mansoura University, Mansoura, Egypt.
Saudi J Kidney Dis Transpl. 2017 Jan-Feb;28(1):141-148. doi: 10.4103/1319-2442.198166.
Marfan syndrome (MFS), the founding member of connective tissue disorder, is an autosomal dominant disease; it is caused by a deficiency of the microfibrillar protein fibrillin-1 (FBN1) and characterized by involvement of three main systems; skeletal, ocular, and cardiovascular. More than one thousand mutations in FBN1 gene on chromosome 15 were found to cause MFS. Nephrotic syndrome (NS) had been described in very few patients with MFS being attributed to membranoproliferative glomerulonephritis secondary to infective endocarditis. Focal segmental glomerulosclerosis (FSGS) had been reported in NS in conjunction with MFS without confirming the diagnosis by mutational analysis of FBN1. We hereby present an Egyptian family with MFS documented at the molecular level; it showed a male proband with NS secondary to FSGS, unfortunately, we failed to make any causal link between FBN dysfunction and FSGS. In this context, we review the spectrum of renal involvements occurring in MFS patients.
马凡综合征(MFS)是结缔组织疾病的首个成员,是一种常染色体显性疾病;它由微原纤维蛋白原纤维蛋白-1(FBN1)缺乏引起,其特征是累及三个主要系统:骨骼、眼部和心血管系统。已发现15号染色体上的FBN1基因有一千多个突变会导致MFS。肾病综合征(NS)在极少数MFS患者中被描述,归因于感染性心内膜炎继发的膜增生性肾小球肾炎。局灶节段性肾小球硬化(FSGS)曾在NS合并MFS的患者中被报道,但未通过FBN1的突变分析来确诊。我们在此展示一个在分子水平确诊为MFS的埃及家族;该家族显示一名男性先证者患有继发于FSGS的NS,遗憾的是,我们未能在FBN功能障碍与FSGS之间建立任何因果联系。在此背景下,我们回顾了MFS患者中出现的肾脏受累情况。