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同一纯合二酰基甘油激酶 ε 突变患者的家庭成员出现不同临床表现:病例报告。

Clinically Different Presentations of Family Members With the Same Homozygote Diacylglycerol Kinase Epsilon Mutation: Case Report.

机构信息

From the Department of Pediatric Nephrology, Dr. Sami Ulus Maternity and Child Health and Diseases Training and Research Hospital, Ankara, Turkey.

出版信息

Exp Clin Transplant. 2022 May;20(Suppl 3):45-48. doi: 10.6002/ect.PediatricSymp2022.O13.

Abstract

Membranoproliferative glomerulonephritis and renal microangiopathies may manifest similar clinical presentations and histology. Many genetic mutations that cause these diseases have been reported. Studies on mutations in the gene encoding diacylglycerol kinase epsilon identified a novel pathophysiologic mechanism leading to atypical hemolytic uremic syndrome and/or membranoproliferative glomerulonephritis. Here, we present the different clinical presentations and treatments in 4 family members who carried the same homozygous diacylglycerol kinase epsilon mutation. The first patient (age 5 years, 3 months old at diagnosis) had nephrotic syndrome. The kidney biopsy was membranoproliferative glomerulonephritis; partial remission was achieved with cyclophosphamide, cyclosporine, and mycophenolate mofetil treatment. The second patient (age 5 years, 7 months at diagnosis) presented with overlapping atypical hemolytic uremic syndrome and membranoproliferative glomerulonephritis. Remission could not be achieved with cyclophosphamide, cyclosporine, and mycophenolate mofetil, and hemodialysis treatment was started. At 10 years from first admission, the patient had end-stage kidney disease, and kidney transplant was performed successfully. The third patient was admitted with the diagnosis of nephrotic syndrome at 13 months of age, kidney biopsy showed membranoproliferative glomerulonephritis, and spontaneous remission developed during followup. He presented with hemolytic uremic syndrome 15 months after the first admission, and dialysis was started. Remission was achieved with plasma infusion and eculizumab treatment. The fourth patient (a 7-month-old boy and brother of patient 3) had no clinical or laboratory findings. All patients had genetic analysis, and mutation in exon 2:c.473G>A(p. W158*) was detected. Our related patients with the same mutation showed different clinical and histological findings. However, we did not observe a clear genotype-phenotype correlation in patients with diacylglycerol kinase epsilon nephropathy, suggesting additional factors mediating phenotypic heterogeneity.

摘要

增生性肾小球肾炎和肾微血管病可能表现出相似的临床特征和组织学表现。许多导致这些疾病的基因突变已经被报道。对编码二酰基甘油激酶 ε 的基因突变的研究发现了一种导致非典型溶血尿毒症综合征和/或增生性肾小球肾炎的新的病理生理机制。在这里,我们介绍了 4 名携带相同同源二酰基甘油激酶 ε 突变的家族成员的不同临床表现和治疗方法。第一个患者(年龄 5 岁 3 个月,诊断时)患有肾病综合征。肾活检显示增生性肾小球肾炎;环磷酰胺、环孢素和霉酚酸酯治疗后部分缓解。第二个患者(年龄 5 岁 7 个月,诊断时)表现为重叠的非典型溶血尿毒症综合征和增生性肾小球肾炎。环磷酰胺、环孢素和霉酚酸酯治疗未能缓解,开始进行血液透析治疗。首次入院 10 年后,患者出现终末期肾病,成功进行了肾移植。第三个患者在 13 个月大时因肾病综合征入院,肾活检显示增生性肾小球肾炎,随访期间自行缓解。首次入院 15 个月后出现溶血尿毒症综合征,开始透析。血浆输注和依库珠单抗治疗后缓解。第四个患者(一个 7 个月大的男孩,是患者 3 的弟弟)没有临床或实验室发现。所有患者均进行了基因分析,发现外显子 2:c.473G>A(p.W158*)突变。我们的相关患者具有相同的突变,表现出不同的临床和组织学发现。然而,我们在二酰基甘油激酶 ε 肾病患者中并未观察到明确的基因型-表型相关性,表明存在其他介导表型异质性的因素。

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