Edmond J Safra Program in Parkinson's Disease, Toronto Western Hospital, Toronto, Ontario, Canada.
Morton and Gloria Shulman Movement Disorders Clinic, Toronto, Ontario, Canada.
Mov Disord. 2018 Aug;33(8):1238-1247. doi: 10.1002/mds.27319. Epub 2018 Feb 13.
Ataxia-telangiectasia is an autosomal recessive neurodegenerative disorder that was initially thought to present exclusively in childhood. With the discovery of the ATM gene, the phenotypic spectrum of the condition has expanded. This review elaborates the expanded phenomenology, including oculomotor apraxia and immunodeficiency, and estimates the presence of each movement disorder feature from previously reported literature. Initial manifestations of Ataxia-telangiectasia include cerebellar symptoms (67%), dystonia (18%), choreoathetosis (10%), and tremor (4%), with parkinsonism and myoclonus not reported as initial features. The prevalence of movement disorders during the course of the disease includes cerebellar symptoms (96%), dystonia (89%), parkinsonism (41%), choreoathetosis (89%), myoclonus (92%), and tremor (74%). Phenomenology and age of onset is modulated by presence of residual ATM kinase activity, with genotypes heavily truncating the ATM protein associated with the most severe phenotypes. Ataxia-telangiectasia commonly results in a spectrum of movement disorders beyond ataxia and telangiectasias. © 2018 International Parkinson and Movement Disorder Society.
共济失调毛细血管扩张症是一种常染色体隐性神经退行性疾病,最初被认为仅在儿童期发病。随着 ATM 基因的发现,该病症的表型谱已经扩大。本综述详细阐述了扩大的表型,包括眼球运动不能和免疫缺陷,并根据之前的文献报道评估了每种运动障碍特征的存在情况。共济失调毛细血管扩张症的初始表现包括小脑症状(67%)、肌张力障碍(18%)、舞蹈手足徐动症(10%)和震颤(4%),帕金森病和肌阵挛并未被报道为初始特征。在疾病过程中运动障碍的发生率包括小脑症状(96%)、肌张力障碍(89%)、帕金森病(41%)、舞蹈手足徐动症(89%)、肌阵挛(92%)和震颤(74%)。残余 ATM 激酶活性的存在会调节表型和发病年龄,ATM 蛋白严重截断的基因型与最严重的表型相关。共济失调毛细血管扩张症通常会导致一系列运动障碍,而不仅仅是共济失调和毛细血管扩张。2018 年国际帕金森病和运动障碍学会。