Sobral Isa, Cachulo Maria da Luz, Figueira João, Silva Rufino
Department of Ophthalmology, Centro Hospitalar e Universitário de Coimbra, Coimbra, Portugal.
BMJ Case Rep. 2014 Oct 16;2014:bcr2014205871. doi: 10.1136/bcr-2014-205871.
Sialidosis is a lysosomal storage disease caused by deficit of neuraminidase. It is an autosomal recessive disease, heterogeneous in its onset, presentation and prognosis. We report a case of a male patient with molecular and enzymatic confirmation of the diagnosis. Symptoms began at age 26 with reduced visual acuity, bilateral cherry-red spots and later myoclonus. A brother, now deceased, had the same confirmed disease. We describe the symptoms and clinical findings of the patient, as well review the current knowledge on the topic. With this report, we highlight the importance of a clinical history integrating all the patient's symptoms in order to achieve the diagnosis. In the presence of a cherry-red spot, a comprehensive study is mandatory. Despite being a rare disease, sialidosis carries a significant burden for its patients and its diagnosis should always be considered in the appropriate setting.
唾液酸沉积症是一种由神经氨酸酶缺乏引起的溶酶体贮积病。它是一种常染色体隐性疾病,在发病、表现和预后方面具有异质性。我们报告一例经分子和酶学确诊的男性患者病例。症状始于26岁,表现为视力下降、双侧樱桃红斑,随后出现肌阵挛。一名现已去世的兄弟也确诊患有相同疾病。我们描述了该患者的症状和临床发现,并回顾了关于该主题的当前知识。通过本报告,我们强调了整合患者所有症状的临床病史对于实现诊断的重要性。在出现樱桃红斑的情况下,必须进行全面检查。尽管唾液酸沉积症是一种罕见疾病,但它给患者带来了巨大负担,在适当情况下应始终考虑对其进行诊断。