Valera Yepes Rocío, Virgili Casas Maria, Povedano Panades Monica, Guerrero Gual Mireia, Villabona Artero Carles
Servicio Endocrinología y Nutrición, Hospital Universitario Bellvitge, L'Hospitalet de Llobregat, Barcelona, España.
Servicio de Neurología, Hospital Universitario Bellvitge, L'Hospitalet de Llobregat, Barcelona, España.
Endocrinol Nutr. 2015 May;62(5):224-30. doi: 10.1016/j.endonu.2015.02.008. Epub 2015 Apr 6.
Kennedy's disease, also known as bulbospinal muscular atrophy, is a rare, X-linked recessive neurodegenerative disorder affecting adult males. It is caused by expansion of an unstable cytosine-adenine-guanine tandem-repeat in exon 1 of the androgen-receptor gene on chromosome Xq11-12, and is characterized by spinal motor neuron progressive degeneration. Endocrinologically, these patients often have the features of hypogonadism associated to the androgen insensitivity syndrome, particularly its partial forms. We report 4 cases with the typical neurological presentation, consisting of slowly progressing generalized muscle weakness with atrophy and bulbar muscle involvement; these patients also had several endocrine manifestations; the most common non-neurological manifestation was gynecomastia. In all cases reported, molecular analysis showed an abnormal cytosine-adenine-guanine triplet repeat expansion in the androgen receptor gene.
肯尼迪病,又称球部脊髓性肌萎缩症,是一种罕见的、X连锁隐性神经退行性疾病,影响成年男性。它由位于Xq11 - 12染色体上雄激素受体基因外显子1中不稳定的胞嘧啶 - 腺嘌呤 - 鸟嘌呤串联重复序列扩增引起,其特征为脊髓运动神经元进行性变性。在内分泌方面,这些患者常具有与雄激素不敏感综合征相关的性腺功能减退特征,尤其是其部分形式。我们报告4例具有典型神经学表现的病例,包括缓慢进展的全身性肌肉无力伴萎缩及延髓肌受累;这些患者还有多种内分泌表现;最常见的非神经学表现是男性乳房发育。在所有报告的病例中,分子分析显示雄激素受体基因存在异常的胞嘧啶 - 腺嘌呤 - 鸟嘌呤三联体重复序列扩增。