Deltas Constantinos, Savva Isavella, Voskarides Konstantinos, Papazachariou Louiza, Pierides Alkis
Molecular Medicine Research Center and Laboratory of Molecular and Medical Genetics, Department of Biological Sciences, University of Cyprus, Nicosia, Cyprus.
Nephron. 2015;130(4):271-80. doi: 10.1159/000435789. Epub 2015 Jul 17.
Collagen IV nephropathies (COL4Ns) comprise benign familial microscopic hematuria, thin basement membrane nephropathy (TBMN), X-linked Alport syndrome (AS) and also autosomal recessive and dominant AS. Apart from the X-linked form of AS, which is caused by hemizygous mutations in the COL4A5 gene, the other entities are caused by mutations in the COL4A3 or COL4A4 genes. The diagnosis of these conditions used to be based on clinical and/or histological findings of renal biopsies, but it is the new molecular genetics approach that revolutionised their investigation and proved particularly instrumental, especially, in many not so clear-cut cases. More recently, the spectrum of COL4N has expanded to include late onset focal segmental glomerulosclerosis (FSGS) that develops on top of TBMN in later life. Also, other reports showed that some patients with a primary diagnosis of familial FSGS proved to have variants in COL4 genes. In the presence of a renal biopsy picture of FSGS and in the absence of either electron microscopy studies or molecular genetic studies that point to TBMN and COL4N, the patient and his family may be mistakenly diagnosed with hereditary FSGS leading to unnecessary further investigations, erroneous family counselling and improper corticosteroid treatment. TBMN is a frequent finding in the general population, and according to several recent reports, it may be the underlying cause and the explanation for many familial and sporadic cases of late-onset FSGS with non-nephrotic proteinuria. This is an important new finding that needs widespread recognition. It is anticipated that the molecular genetic analysis with next generation sequencing will certainly offer timely correct diagnosis.
IV型胶原肾病(COL4Ns)包括良性家族性镜下血尿、薄基底膜肾病(TBMN)、X连锁遗传性肾炎(AS)以及常染色体隐性和显性AS。除了由COL4A5基因半合子突变引起的X连锁形式的AS外,其他类型是由COL4A3或COL4A4基因的突变引起的。这些疾病的诊断过去基于肾活检的临床和/或组织学结果,但新的分子遗传学方法彻底改变了对它们的研究,并且在许多不那么明确的病例中证明特别有用。最近,COL4N的范围已经扩大,包括在晚年TBMN基础上发生的迟发性局灶节段性肾小球硬化(FSGS)。此外,其他报告显示,一些最初诊断为家族性FSGS的患者被证明存在COL4基因变异。在存在FSGS的肾活检表现且没有电子显微镜研究或分子遗传学研究指向TBMN和COL4N的情况下,患者及其家人可能会被误诊为遗传性FSGS,从而导致不必要的进一步检查、错误的遗传咨询和不适当的皮质类固醇治疗。TBMN在普通人群中很常见,根据最近的几份报告,它可能是许多伴有非肾病性蛋白尿的迟发性FSGS家族性和散发性病例的潜在原因及解释。这是一项需要广泛认可的重要新发现。预计下一代测序的分子遗传学分析肯定会提供及时的正确诊断。