Nakaoka Sachiko, Kondo Hidehito, Matsuoka Keiko, Shibuya Toko, Otomo Takanobu, Hamada Yusuke, Sakamoto Kenichi, Ozono Keiichi, Sakai Norio
Department of Pediatrics, Osaka University Graduate School of Medicine, Japan; Department of Pediatrics, Faculty of Medicine, University of Toyama, Japan.
Department of Pediatrics, Osaka University Graduate School of Medicine, Japan; Department of Pediatrics and Neonatology, Japanese Red Cross Kyoto Daiichi Hospital, Japan.
Brain Dev. 2021 Sep;43(8):867-872. doi: 10.1016/j.braindev.2021.04.003. Epub 2021 May 6.
In mucopolysaccharidoses (MPS), spinal cord compression (SCC) resulting from glycosaminoglycan (GAG) accumulation is a critical complication that can cause significant neurological and respiratory morbidities. However, clinically similar disorders such as mucolipidosis types II and III (ML) with SCC have been scarcely reported. Herein, we report four patients with ML who had SCC. Brain MRI revealed progressive spinal canal stenosis and SCC. In addition, T2-weighted high signal changes in the cervical cord were detected in two cases. Severe cases of SCC were detected as early as 1 year of age. All cases had respiratory problems. One case showed severe hypoxia and another, severe sleep apnea. In two cases, respiratory insufficiency and tetraplegia rapidly progressed as SCC progressed. Then, the patients became bedridden and needed artificial ventilation. In addition, two of the four patients died of respiratory failure. The autopsy of one patient revealed a compressed cervical cord and marked dura mater thickening due to GAG accumulation. These findings suggest that the accumulation of substrates in the dura mater caused SCC in the patients with ML. Our cases indicate that SCC is expected to be a common and critical complication of ML and MPS. MRI evaluation of cervical involvements and careful clinical observation are required in patients with ML.
在黏多糖贮积症(MPS)中,糖胺聚糖(GAG)蓄积导致的脊髓压迫(SCC)是一种关键并发症,可引起严重的神经和呼吸疾病。然而,临床上类似的疾病如伴有SCC的II型和III型黏脂贮积症(ML)鲜有报道。在此,我们报告4例患有SCC的ML患者。脑部磁共振成像(MRI)显示椎管进行性狭窄和SCC。此外,2例患者检测到颈髓T2加权高信号改变。严重的SCC病例早在1岁时就被检测到。所有病例均有呼吸问题。1例表现为严重缺氧,另1例表现为严重睡眠呼吸暂停。2例患者中,随着SCC进展,呼吸功能不全和四肢瘫痪迅速加重。随后,患者卧床不起,需要人工通气。此外,4例患者中有2例死于呼吸衰竭。1例患者的尸检显示颈髓受压,由于GAG蓄积导致硬脑膜明显增厚。这些发现表明,硬脑膜中底物的蓄积导致了ML患者的SCC。我们的病例表明,SCC预计是ML和MPS的常见且关键的并发症。ML患者需要进行颈椎受累情况的MRI评估和仔细的临床观察。