Sanefuji Masafumi, Kira Ryutaro, Matsumoto Kenichi, Gondo Kenjiro, Torisu Hiroyuki, Kawakami Hideshi, Iwaki Toru, Hara Toshiro
Department of Pediatrics, Graduate School of Medical Sciences, Kyushu University, Higashi-ku, Fukuoka, Japan.
J Child Neurol. 2010 Nov;25(11):1429-34. doi: 10.1177/0883073810372991. Epub 2010 Jun 17.
The combination of pontocerebellar hypoplasia and anterior horn cell degeneration is classified as pontocerebellar hypoplasia type 1. Although most cases exhibit severe muscle weakness and hypotonia neonatally with short life spans, some cases exhibit a later onset with a longer life span and show cerebellar atrophy without pontine involvement. We present a child who exhibited neurological deterioration and progressive atrophy of the cerebellum and pons, with onset of symptoms at 20 months and death at 15 years of age. The pathological findings disclosed anterior horn cell degeneration and pyramidal tract involvement in addition to pontocerebellar atrophy, leading to the diagnosis of pontocerebellar hypoplasia type 1. The present case suggests that the degenerative pattern of later-onset pontocerebellar hypoplasia type 1 is similar to that of prenatal-onset cases. Further reports of later-onset cases with histopathological examination are required to elucidate the nosology and etiology of the disorder.
桥脑小脑发育不全与前角细胞变性的组合被归类为1型桥脑小脑发育不全。虽然大多数病例在新生儿期表现出严重的肌肉无力和肌张力减退,寿命较短,但有些病例起病较晚,寿命较长,表现为小脑萎缩而无桥脑受累。我们报告一名儿童,其出现神经功能恶化以及小脑和桥脑进行性萎缩,症状在20个月时出现,15岁时死亡。病理检查结果除桥脑小脑萎缩外,还发现前角细胞变性和锥体束受累,从而诊断为1型桥脑小脑发育不全。本病例提示,晚发型1型桥脑小脑发育不全的退变模式与产前发病的病例相似。需要进一步报告晚发型病例并进行组织病理学检查,以阐明该疾病的分类学和病因。