Institute of Medical & Molecular Genetics (INGEMM), Hospital Universitario La Paz, Universidad Autónoma de Madrid, IdiPAZ, Madrid, Spain.
Department of Pediatrics, Hospital Universitario la Paz, Madrid, Spain.
Am J Med Genet A. 2021 Mar;185(3):856-865. doi: 10.1002/ajmg.a.62010. Epub 2020 Dec 11.
Stuve-Wiedemann syndrome (SWS; MIM 601559) is a rare autosomal recessive disease caused by mutations in the leukemia inhibitor factor receptor gene (LIFR). Common clinical and radiological findings are often observed, and high neonatal mortality occurs due to respiratory distress and hyperthermic episodes. Despite initially considered as a lethal disorder during the newborn period, in recent years, several SWS childhood survivors have been reported. We report a detailed clinical and radiological characterization of four unrelated childhood SWS molecularly confirmed patients and review 22 previously reported childhood surviving cases. We contribute to the definition of the childhood survival phenotype of SWS, emphasizing the evolving phenotype, characterized by skeletal abnormalities with typical radiological findings, distinctive dysmorphic features, and dysautonomia. Based on the typical features and clinical course, early diagnosis is possible and crucial to plan appropriate management and prevent potential complications. Genetic confirmation is advisable in order to improve genetic counseling to the patients and their families.
斯图-威德曼综合征(SWS;MIM 601559)是一种罕见的常染色体隐性疾病,由白血病抑制因子受体基因(LIFR)的突变引起。常见的临床和影像学表现经常观察到,由于呼吸窘迫和发热发作,新生儿死亡率很高。尽管最初在新生儿期被认为是一种致命疾病,但近年来,已有几例 SWS 儿童幸存者被报道。我们报告了四个无关联的分子确诊的儿童 SWS 患者的详细临床和影像学特征,并回顾了 22 例先前报道的儿童存活病例。我们为 SWS 的儿童存活表型的定义做出了贡献,强调了不断演变的表型,其特征是骨骼异常伴典型的影像学表现、独特的畸形特征和自主神经功能障碍。基于典型的特征和临床过程,早期诊断是可能的,对于规划适当的管理和预防潜在的并发症至关重要。遗传确认是可取的,以改善对患者及其家属的遗传咨询。