Lo Raymond Y, Figueroa Karla P, Pulst Stefan M, Lin Chi-Ying, Perlman Susan, Wilmot George, Gomez Christopher, Schmahmann Jeremy, Paulson Henry, Shakkottai Vikram G, Ying Sarah, Zesiewicz Theresa, Bushara Khalaf, Geschwind Michael, Xia Guangbin, Subramony S H, Ashizawa Tetsuo, Kuo Sheng-Han
Department of Neurology, Buddhist Tzu Chi General Hospital and Tzu Chi University, Hualien, Taiwan.
Mov Disord. 2015 Feb;30(2):214-20. doi: 10.1002/mds.26088. Epub 2014 Dec 1.
The aim of this study was to investigate the association between drug exposure and disease severity in SCA types 1, 2, 3 and 6. The Clinical Research Consortium for Spinocerebellar Ataxias (CRC-SCA) enrolled 319 participants with SCA1, 2, 3, and 6 from 12 medical centers in the United States and repeatedly measured clinical severity by the Scale for Assessment and Rating of Ataxia (SARA), the Unified Huntington's Disease Rating Scale part IV (UHDRS-IV), and the 9-item Patient Health Questionnaire during July 2009 to May 2012. We employed generalized estimating equations in regression models to study the longitudinal effects of coenzyme Q10 (CoQ10), statin, and vitamin E on clinical severity of ataxia after adjusting for age, sex, and pathological CAG repeat number. Cross-sectionally, exposure to CoQ10 was associated with lower SARA and higher UHDRS-IV scores in SCA1 and 3. No association was found between statins, vitamin E, and clinical outcome. Longitudinally, CoQ10, statins, and vitamin E did not change the rates of clinical deterioration indexed by SARA and UHDRS-IV scores within 2 years. CoQ10 is associated with better clinical outcome in SCA1 and 3. These drug exposures did not appear to influence clinical progression within 2 years. Further studies are warranted to confirm the association.
本研究的目的是调查1型、2型、3型和6型脊髓小脑共济失调（SCA）中药物暴露与疾病严重程度之间的关联。脊髓小脑共济失调临床研究联盟（CRC-SCA）从美国12个医疗中心招募了319名患有SCA1、2、3和6型的参与者，并在2009年7月至2012年5月期间，通过共济失调评估和评级量表（SARA）、统一亨廷顿舞蹈病评定量表第四部分（UHDRS-IV）以及9项患者健康问卷反复测量临床严重程度。我们在回归模型中采用广义估计方程，在调整年龄、性别和病理性CAG重复次数后，研究辅酶Q10（CoQ10）、他汀类药物和维生素E对共济失调临床严重程度的纵向影响。横断面研究中，在SCA1和3型中，CoQ10暴露与较低的SARA评分和较高的UHDRS-IV评分相关。未发现他汀类药物、维生素E与临床结局之间存在关联。纵向研究中，CoQ10、他汀类药物和维生素E在2年内并未改变以SARA和UHDRS-IV评分作为指标的临床恶化率。CoQ10与SCA1和3型更好的临床结局相关。这些药物暴露在2年内似乎并未影响临床进展。有必要进行进一步研究以证实这种关联。
