Kungurtseva A L, Popovich A V, Tikhonovich Y V, Vitebskaya A V
I.M. Sechenov First Moscow State Medical University (Sechenov University).
Probl Endokrinol (Mosk). 2023 Oct 8;70(2):86-93. doi: 10.14341/probl13369.
Wiedemann-Rautenstrauch syndrome (neonatal progeroid syndrome) is an ultra-orphan disease from the group of premature aging syndromes with an autosomal recessive type of inheritance associated with mutations in the POLR3A, POLR3B, and POLR3GL genes encoding RNA polymerase III. The incidence of the disease is currently unknown. We present the first clinical description in Russian Federation of a patient 7 years 6 months old with Wiedemann-Rautenstrauch syndrome (compound heterozygous mutations in POLR3A gene) with progeroid features, adentia, growth retardation (height SDS -3,41, height velocity SDS -2,47), underweight (BMI SDS -6,20), and generalized lipodystrophy. The article presents the observation of the patient for 1.5 years, the world experience of dynamic follow-up of patients with neonatal progeroid syndrome, differential diagnosis, as well as recommendations for the management of patients with this syndrome. Given the lack of specific treatment to date, patients are observed by a multidisciplinary team of physicians.
维德曼-劳滕施劳赫综合征(新生儿早老综合征)是早老综合征中的一种极为罕见的疾病,具有常染色体隐性遗传类型,与编码RNA聚合酶III的POLR3A、POLR3B和POLR3GL基因突变相关。该疾病的发病率目前尚不清楚。我们首次在俄罗斯联邦对一名7岁6个月大的维德曼-劳滕施劳赫综合征患者(POLR3A基因复合杂合突变)进行了临床描述,该患者具有早老特征、无牙症、生长发育迟缓(身高标准差评分-3.41,身高增长速度标准差评分-2.47)、体重不足(体重指数标准差评分-6.20)以及全身性脂肪营养不良。本文介绍了对该患者1.5年的观察情况、新生儿早老综合征患者动态随访的全球经验、鉴别诊断以及对该综合征患者管理的建议。鉴于目前缺乏特效治疗方法,患者由多学科医生团队进行观察。