Richard D. Wood Jr. Center for Fetal Diagnosis and Treatment, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Department of Surgery, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA.
Prenat Diagn. 2024 May;44(5):665-668. doi: 10.1002/pd.6563. Epub 2024 Mar 30.
Oculocerebrorenal syndrome (Lowe syndrome) is a rare X-linked disorder affecting 1/500,000 males that most frequently affects the eyes, central nervous system, and kidneys. Phenotypic presentation includes congenital cataracts, developmental delay, intellectual disability, and Fanconi-type renal dysfunction. Lowe Syndrome is caused by hemizygous loss of function variants in the OCRL gene. While individuals may live into the third and fourth decade of life, some will die in the first few years of either renal failure or infection. While early diagnosis is important, few cases have documented the prenatal phenotype of this condition, which has included bilateral cataracts and variable neurological abnormalities. We report a case of a family with an extensive history of congenital cataracts, immune compromise, and neonatal death in male members. The fetus was found to have a unilateral cataract, mild ventriculomegaly, vertebral anomalies, and an underlying diagnosis of Lowe Syndrome with a mutation in OCRL at c.2582-1G>C (IVS23-1G>C).
眼脑肾综合征(Lowe 综合征)是一种罕见的 X 连锁疾病,影响 50 万男性中的 1 人,主要影响眼睛、中枢神经系统和肾脏。表型表现包括先天性白内障、发育迟缓、智力残疾和 Fanconi 型肾功能障碍。Lowe 综合征是由 OCRL 基因的杂合功能丧失变异引起的。虽然个体可能会活到第三和第四个十年,但有些人会因肾衰竭或感染而在生命的头几年死亡。早期诊断很重要,但很少有病例记录了这种情况的产前表型,包括双侧白内障和各种神经发育异常。我们报告了一个家族的病例,该家族有先天性白内障、免疫功能低下和男性新生儿死亡的病史。该胎儿被发现患有单侧白内障、轻度脑室扩张、椎体异常,并通过在 OCRL 基因 c.2582-1G>C(IVS23-1G>C)处的突变,确诊为 Lowe 综合征。