Yoshimura Ayumi, Kibe Tetsuya, Irahara Kaori, Sakai Norio, Yokochi Kenji
Department of Pediatrics and Pediatric Neurology, Seirei-Mikatahara General Hospital, Shizuoka, Japan.
Department of Pediatrics, Graduate School of Medicine, Osaka University, Osaka, Japan.
Jpn Clin Med. 2016 Sep 18;7:23-6. doi: 10.4137/JCM.S40470. eCollection 2016.
A case of late-infantile Krabbe disease in a patient who presented with developmental regression and spastic quadriplegia in late infancy is reported. Brain magnetic resonance imaging (MRI) at 11 months of age showed predominant corticospinal tract involvement, which usually appears in adult Krabbe disease. Galactocerebrosidase activity in lymphocytes and skin fibroblasts was very low. Genetic testing revealed compound heterozygous mutations of the galactocerebrosidase (GALC) gene, c.635_646 delinsCTC and c.1901T>C [p.L618S], both of which are known pathogenic mutations. It has been reported that the c.1901T>C [p.L618S] mutation is associated with the late-onset phenotype and, in a past case, a homozygous mutation at this location showed predominant corticospinal tract involvement on MRI. Although further analysis is needed to identify the pathophysiological mechanism, this combination of mutations is likely to be associated with this unusual MRI finding in late-infantile Krabbe disease. Because these types of mutations are common for Japanese patients, it is possible that there are more undiagnosed and late-diagnosed patients of late-infantile Krabbe disease who display limited lesions on MRI. Pediatricians should be aware that patients with late-infantile Krabbe disease can present with predominant corticospinal tract involvement on MRI.
报告了1例晚期婴儿型克拉伯病患者,该患者在婴儿晚期出现发育倒退和痉挛性四肢瘫。11个月大时的脑磁共振成像(MRI)显示主要累及皮质脊髓束,这通常见于成人克拉伯病。淋巴细胞和皮肤成纤维细胞中的半乳糖脑苷脂酶活性非常低。基因检测发现半乳糖脑苷脂酶(GALC)基因存在复合杂合突变,即c.635_646 delinsCTC和c.1901T>C [p.L618S],这两种都是已知的致病突变。据报道,c.1901T>C [p.L618S]突变与迟发型表型相关,在过去1例病例中,该位点的纯合突变在MRI上显示主要累及皮质脊髓束。虽然需要进一步分析以确定病理生理机制,但这种突变组合可能与晚期婴儿型克拉伯病这种不寻常的MRI表现有关。由于这些类型的突变在日本患者中很常见,有可能存在更多未诊断和诊断较晚的晚期婴儿型克拉伯病患者,他们在MRI上显示的病变有限。儿科医生应意识到,晚期婴儿型克拉伯病患者在MRI上可能主要表现为皮质脊髓束受累。