Albuquerque Marcus Vinicius Cristino de, Pedroso José Luiz, Braga Neto Pedro, Barsottini Orlando Graziani Povoas
Departamento de Neurologia, Divisão de Neurologia Geral e Ataxias, Universidade Federal de São Paulo, Sao Paulo, SP, Brazil.
Arq Neuropsiquiatr. 2015 Jan;73(1):18-21. doi: 10.1590/0004-282X20140192. Epub 2015 Jan 1.
The spinocerebellar ataxias (SCA) are a group of neurodegenerative disorders characterized by heterogeneous clinical presentation. Spinocerebellar ataxia type 7 (SCA7) is caused by an abnormal CAG repeat expansion and includes cerebellar signs associated with visual loss and ophthalmoplegia. Marked anticipation and dynamic mutation is observed in SCA7. Moreover, phenotype variability and very early onset of symptoms may occur. In this article, a large series of Brazilian patients with different SCA subtypes was evaluated, and we compared the age of onset of SCA7 with other SCA. From the 26 patients with SCA7, 4 manifested their symptoms before 10-year-old. Also, occasionally the parents may have the onset of symptoms after their children. In conclusion, our study highlights the genetic anticipation phenomenon that occurs in SCA7 families. Patients with very early onset ataxia in the context of a remarkable family history, must be considered and tested for SCA7.
脊髓小脑共济失调(SCA)是一组以临床表现异质性为特征的神经退行性疾病。7型脊髓小脑共济失调(SCA7)由异常的CAG重复扩增引起,包括与视力丧失和眼肌麻痹相关的小脑体征。在SCA7中观察到明显的遗传早现和动态突变。此外,可能会出现表型变异性和非常早的症状发作。在本文中,我们评估了一大系列不同SCA亚型的巴西患者,并比较了SCA7与其他SCA的发病年龄。在26例SCA7患者中,4例在10岁之前出现症状。此外,偶尔父母可能在其子女之后出现症状。总之,我们的研究突出了SCA7家族中发生的遗传早现现象。对于有明显家族史且共济失调发病非常早的患者,必须考虑进行SCA7检测。