Kiem Hao Tran, Diem Chi Nguyen Thi, Hong Duc Nguyen Thi, Kim Hoa Nguyen Thi
Pediatric Center, Hue Central Hospital, Hue City, Vietnam.
SAGE Open Med Case Rep. 2020 Jun 29;8:2050313X20938245. doi: 10.1177/2050313X20938245. eCollection 2020.
Mucopolysaccharidosis is a group of rare metabolic disorders characterized by a deficiency of enzymes in the degradation of glycosaminoglycans. The incomplete degradation process leads to the accumulation of glycosaminoglycans in lysosomes of various tissues, which interferes with cell function. We report three cases that were classified as Hurler-Mucopolysaccharidosis I, Morquio-Mucopolysaccharidosis IV A, and Maroteaux-Lamy-Mucopolysaccharidosis VI. Clinical presentations of these cases vary, depending on each type of enzyme defect. All the patients appeared healthy at birth, and symptoms appear at around 1 or 2 years. Clinical features, radiological findings, and especially enzyme assays have allowed us to establish a definitive diagnosis in these cases. These cases highlight that abnormal clinical symptoms, such as growth failure, coarse facial features, and joint problems, are key points for further investigation relating to mucopolysaccharidosis disease. However, in low- and middle-income countries, it is difficult to have a definitive diagnosis of one of the mucopolysaccharidoses due to lacking enzyme assays.
黏多糖贮积症是一组罕见的代谢性疾病,其特征是糖胺聚糖降解过程中酶缺乏。不完全的降解过程导致糖胺聚糖在各种组织的溶酶体中积累,从而干扰细胞功能。我们报告了三例分别被归类为I型Hurler黏多糖贮积症、IV A型Morquio黏多糖贮积症和VI型Maroteaux-Lamy黏多糖贮积症的病例。这些病例的临床表现各不相同,取决于每种类型的酶缺陷。所有患者出生时看起来健康,症状大约在1或2岁时出现。临床特征、影像学检查结果,尤其是酶分析使我们能够对这些病例做出明确诊断。这些病例表明,生长发育迟缓、面部粗糙和关节问题等异常临床症状是进一步调查黏多糖贮积症疾病的关键点。然而,在低收入和中等收入国家,由于缺乏酶分析,很难对黏多糖贮积症之一做出明确诊断。