Department of Pediatrics, Kobe University Graduate School of Medicine, 7-5-1 Kusunoki-cho, Chuo-ku, Kobe, 650-0017, Japan.
Division of Pediatric Nephrology, Okinawa Prefectural Nanbu Medical Center, Children's Medical Center, 118-1 Arakawa, Haebaru-cho, Simajiri-gun, Okinawa, 901-1105, Japan.
CEN Case Rep. 2020 Nov;9(4):431-436. doi: 10.1007/s13730-020-00503-8. Epub 2020 Jul 4.
X-linked Alport syndrome (XLAS) is a progressive hereditary kidney disease caused by mutations in the COL4A5 gene encoding the type IV collagen α5 chain. To date, 11 cases having somatic mosaic variants in COL4A5 have been reported; however, all of them involved single-nucleotide variations (SNVs). Here, we report a female XLAS patient with somatic mosaicism identified by copy number variation (CNV) in COL4A5. The case was a 35-year-old female, the mother of the proband, whose only clinical symptom was hematuria. The proband, who was the son of this patient, was diagnosed with XLAS by gene testing, which showed a large hemizygous deletion from exon 3-51 in COL4A5 detected by next-generation sequencing and then confirmed by multiplex ligation-dependent probe amplification (MLPA). Then, MLPA analysis revealed that the female patient had the same deletion with only a 20% copy number reduction compared with a normal female control; she was thus diagnosed with XLAS with somatic mosaicism. CNVs in COL4A5 are relatively rare and, to the best of our knowledge, somatic mosaic variants with CNVs have never been reported. This case clearly featured a germline variant because the patient's son exhibited XLAS. This is thus the first case report on an XLAS patient having CNV in COL4A5 with somatic mosaicism. The obtained findings were very important for the genetic counseling of this family.
X 连锁 Alport 综合征(XLAS)是一种渐进性遗传性肾脏疾病,由编码 IV 型胶原 α5 链的 COL4A5 基因突变引起。迄今为止,已有 11 例 COL4A5 中存在体细胞镶嵌变异的病例报告;然而,它们都涉及单核苷酸变异(SNVs)。在这里,我们报告了一例通过 COL4A5 拷贝数变异(CNV)鉴定的女性 XLAS 患者的体细胞镶嵌现象。该病例为一名 35 岁女性,为先证者的母亲,唯一的临床症状是血尿。先证者是该患者的儿子,通过基因检测被诊断为 XLAS,这表明通过下一代测序检测到 COL4A5 外显子 3-51 处存在大片段纯合缺失,随后通过多重连接依赖性探针扩增(MLPA)得到了证实。然后,MLPA 分析显示,与正常女性对照相比,该女性患者的同一缺失仅减少了 20%的拷贝数,因此被诊断为具有体细胞镶嵌现象的 XLAS。COL4A5 中的 CNVs 相对较少,据我们所知,尚未有关于 COL4A5 体细胞镶嵌变异的 CNVs 的报道。该病例明显具有种系变异,因为患者的儿子表现出 XLAS。这是首例关于 COL4A5 中存在 CNV 并具有体细胞镶嵌现象的 XLAS 患者的病例报告。所得结果对该家系的遗传咨询非常重要。